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Posts by DMD WarrioR

SonoThera’s RIPPLE Technology Offers Hope for Full-Length Human Dystrophin SonoThera, a biotechnology company dedicated to treat the root causes of human diseases by developing the next generation of genetic medicines, announced on January 8, 2026, that its proprietary RIPPLE™ technology had detected strong and persistent full-length human dystrophin protein expression in the skeletal muscles of non-human primates, reaching up to 290% of normal levels. To learn more about SonoThera’s RIPPLE technology firsthand, we contacted Elizabeth Harness from the Corporate Communications department and shared our questions on behalf of our followers. Elizabeth thoughtfully and comprehensively provided details about Ultrasound-Mediated Delivery (UMD) technology, which is also planned for use in Duchenne muscular dystrophy.

SonoThera’s RIPPLE Technology Offers Hope for Full-Length Human Dystrophin SonoThera, a biotechnology company dedicated to treat the root causes of human diseases by developing the next generation of genetic medicines, announced on January 8, 2026, that its proprietary RIPPLE™ technology had detected strong and persistent full-length human dystrophin protein expression in the skeletal muscles of non-human primates, reaching up to 290% of normal levels. To learn more about SonoThera’s RIPPLE technology firsthand, we contacted Elizabeth Harness from the Corporate Communications department and shared our questions on behalf of our followers. Elizabeth thoughtfully and comprehensively provided details about Ultrasound-Mediated Delivery (UMD) technology, which is also planned for use in Duchenne muscular dystrophy.

SonoThera’s RIPPLE Technology Offers Hope for Full-Length Human Dystrophin

👉 Read More: DMDWarrioR.com

2 months ago 1 0 0 0
Ractigen Therapeutics Doses First Patient in IIT Study of RAG-18, a Potential Game-Changing #saRNA Therapeutic for #Duchenne Muscular Dystrophy Ractigen Therapeutics, a pioneering developer of small activating RNA (saRNA) therapeutics, today announced the successful dosing of the first patient in its investigator-initiated trial (IIT) of RAG-18 at Peking Union Medical College Hospital (PUMCH), Chinese Academy of Medical Sciences in Beijing. #DMD 👉 Read More: DMDWarrioR.com

Ractigen Therapeutics Doses First Patient in IIT Study of RAG-18, a Potential Game-Changing #saRNA Therapeutic for #Duchenne Muscular Dystrophy Ractigen Therapeutics, a pioneering developer of small activating RNA (saRNA) therapeutics, today announced the successful dosing of the first patient in its investigator-initiated trial (IIT) of RAG-18 at Peking Union Medical College Hospital (PUMCH), Chinese Academy of Medical Sciences in Beijing. #DMD 👉 Read More: DMDWarrioR.com

Ractigen Therapeutics Doses First Patient in IIT Study of RAG-18, a Potential Game-Changing #saRNA Therapeutic for #Duchenne Muscular Dystrophy

👉 Read More: DMDWarrioR.com

3 months ago 1 0 0 0
Atossa Therapeutics Receives FDA Rare Pediatric Disease Designation for (Z)-Endoxifen for Duchenne Muscular Dystrophy Atossa Therapeutics announced that the U.S. Food and Drug Administration (“FDA”) has granted Rare Pediatric Disease (“RPD”) designation to (Z)-Endoxifen for the treatment of Duchenne Muscular Dystrophy (“DMD”). 👉 Read More: DMDWarrioR.com

Atossa Therapeutics Receives FDA Rare Pediatric Disease Designation for (Z)-Endoxifen for Duchenne Muscular Dystrophy Atossa Therapeutics announced that the U.S. Food and Drug Administration (“FDA”) has granted Rare Pediatric Disease (“RPD”) designation to (Z)-Endoxifen for the treatment of Duchenne Muscular Dystrophy (“DMD”). 👉 Read More: DMDWarrioR.com

Atossa Therapeutics Receives FDA Rare Pediatric Disease Designation for (Z)-Endoxifen for Duchenne Muscular Dystrophy

👉 Read More: DMDWarrioR.com

3 months ago 0 0 0 0
Satellos Receives FDA and International Clearance to Commence Pediatric Phase 2 Testing of SAT-3247 for Duchenne Muscular Dystrophy Satellos reported that the FDA and other global regulators have cleared its IND for SAT-3247, enabling a three-month Phase 2, placebo-controlled trial in 51 ambulatory children with Duchenne muscular dystrophy. #satellos #dmd #duchenne 👉 Read More: DMDWarrioR.com

Satellos Receives FDA and International Clearance to Commence Pediatric Phase 2 Testing of SAT-3247 for Duchenne Muscular Dystrophy Satellos reported that the FDA and other global regulators have cleared its IND for SAT-3247, enabling a three-month Phase 2, placebo-controlled trial in 51 ambulatory children with Duchenne muscular dystrophy. #satellos #dmd #duchenne 👉 Read More: DMDWarrioR.com

Satellos reported that the FDA and other global regulators have cleared its IND for SAT-3247, enabling a three-month Phase 2, placebo-controlled trial in 51 ambulatory children with Duchenne muscular dystrophy. #satellos #dmd #duchenne

👉 Read More: DMDWarrioR.com

3 months ago 0 0 0 0
Researchers Develop Optimized Smad7 Gene Therapy to Target Duchenne Muscular Dystrophy Researchers Buel D. Rodgers and Christopher W. Ward published a groundbreaking study in Gene Therapy that unveiled a codon-optimized human Smad7 gene therapy aimed at treating Duchenne muscular dystrophy (DMD). 👉 Read More: DMDWarrioR.com

Researchers Develop Optimized Smad7 Gene Therapy to Target Duchenne Muscular Dystrophy Researchers Buel D. Rodgers and Christopher W. Ward published a groundbreaking study in Gene Therapy that unveiled a codon-optimized human Smad7 gene therapy aimed at treating Duchenne muscular dystrophy (DMD). 👉 Read More: DMDWarrioR.com

Researchers Buel D. Rodgers and Christopher W. Ward published a groundbreaking study in Gene Therapy that unveiled a codon-optimized human Smad7 gene therapy aimed at treating Duchenne muscular dystrophy (DMD).

👉 Read More: DMDWarrioR.com

4 months ago 0 0 0 0
ELEVIDYS Gene Therapy for Non-Ambulant Children with DMD: FDA OKs ENDEAVOR Cohort 8 Immunosuppression Study

ELEVIDYS Gene Therapy for Non-Ambulant Children with DMD: FDA OKs ENDEAVOR Cohort 8 Immunosuppression Study

ELEVIDYS Gene Therapy for Non-Ambulant Children with DMD: FDA OKs ENDEAVOR Cohort 8 Immunosuppression Study

👉 Read More: DMDWarrioR.com

4 months ago 0 0 0 0
Potential of SAT-3247 to Restore Muscle Regeneration in Duchenne Published in Nature Satellos Therapeutics announced that researchers at the Ottawa Hospital Research Institute have published new findings in Nature Communications validating the company’s innovative approach to treating the root cause of Duchenne muscular dystrophy and related diseases. 👉 Read More: DMDWarrioR.com

Potential of SAT-3247 to Restore Muscle Regeneration in Duchenne Published in Nature Satellos Therapeutics announced that researchers at the Ottawa Hospital Research Institute have published new findings in Nature Communications validating the company’s innovative approach to treating the root cause of Duchenne muscular dystrophy and related diseases. 👉 Read More: DMDWarrioR.com

Potential of SAT-3247 to Restore Muscle Regeneration in Duchenne Published in Nature

👉 Read More: DMDWarrioR.com

4 months ago 0 0 0 0
Ali Taghizadeh Announces DMD ANKA mRNA Therapy, Currently in Clinical Trial in Turkiye DMD ANKA mRNA therapy, currently under development for Duchenne muscular dystrophy in Turkiye, was announced by Associate Professor Dr. Ali Taghizadeh.

Ali Taghizadeh Announces DMD ANKA mRNA Therapy, Currently in Clinical Trial in Turkiye DMD ANKA mRNA therapy, currently under development for Duchenne muscular dystrophy in Turkiye, was announced by Associate Professor Dr. Ali Taghizadeh.

DMD ANKA mRNA therapy, currently under development for Duchenne muscular dystrophy in Turkiye, was announced by Associate Professor Dr. Ali Taghizadeh.

👉 Read More: DMDWarrioR.com

4 months ago 0 0 0 0
REGENXBIO Announces Completion of RGX-202 Pivotal Enrollment and Initiates Commercial Production in Duchenne Gene Therapy Program REGENXBIO announced it has completed enrollment of 30 participants in the pivotal portion of its AFFINITY DUCHENNE trial for RGX-202, an investigational gene therapy for Duchenne muscular dystrophy.

REGENXBIO Announces Completion of RGX-202 Pivotal Enrollment and Initiates Commercial Production in Duchenne Gene Therapy Program REGENXBIO announced it has completed enrollment of 30 participants in the pivotal portion of its AFFINITY DUCHENNE trial for RGX-202, an investigational gene therapy for Duchenne muscular dystrophy.

REGENXBIO Announces Completion of RGX-202 Pivotal Enrollment and Initiates Commercial Production in Duchenne Gene Therapy Program

#dmd #duchenne #regenxbio #genetherapy #clinicaltrials #rgx202

👉 Read More: DMDWarrioR.com

5 months ago 0 0 0 0
Hansa’ Imlifidase Reduce Anti-AAV Antibodies and Allow Administration of Gene Therapy Two gene therapy trials, one sponsored by Sarepta and the other sponsored by Genethon, support the ability of imlifidase to substantially reduce anti-AAV antibodies to allow administration of gene therapy.

Hansa’ Imlifidase Reduce Anti-AAV Antibodies and Allow Administration of Gene Therapy Two gene therapy trials, one sponsored by Sarepta and the other sponsored by Genethon, support the ability of imlifidase to substantially reduce anti-AAV antibodies to allow administration of gene therapy.

Hansa’ Imlifidase Reduce Anti-AAV Antibodies and Allow Administration of Gene Therapy

#dmd #duchenne #imlifidase #genetherapy #clinicaltrials

👉 Read More: DMDWarrioR.com

5 months ago 0 0 0 0
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If you're searching for answers to 'why Elevidys was not approved by the European Medicines Agency (EMA)', you're not alone. In this article, we explore the key reasons behind the EMA’s decision and what it means for patients, families, and the future of gene therapy in the EU.

If you're searching for answers to 'why Elevidys was not approved by the European Medicines Agency (EMA)', you're not alone. In this article, we explore the key reasons behind the EMA’s decision and what it means for patients, families, and the future of gene therapy in the EU.

Why Elevidys Was Not Approved by the European Medicines Agency (EMA)?

You can access the comprehensive report on the reasons for the Elevidys refusal published by EMA from our web page link.

#dmd #duchenne #elevidys #eu

👉 Read More: DMDWarrioR.com

5 months ago 0 0 0 0
Brogidirsen (NS-089/NCNP-02) has The Potential to Slow Disease Progression in DMD Patients Amenable to Exon 44 Skipping Nippon Shinyaku announced efficacy and safety data from a 3.5-year application based on an open-label extension study that also included the initiation of a clinical trial for brogidirsen (NS-089/NCNP-02). #dmd #duchenne #exon44 #brogidirsen 👉 Read More: DMDWarrioR.com

Brogidirsen (NS-089/NCNP-02) has The Potential to Slow Disease Progression in DMD Patients Amenable to Exon 44 Skipping Nippon Shinyaku announced efficacy and safety data from a 3.5-year application based on an open-label extension study that also included the initiation of a clinical trial for brogidirsen (NS-089/NCNP-02). #dmd #duchenne #exon44 #brogidirsen 👉 Read More: DMDWarrioR.com

Brogidirsen (NS-089/NCNP-02) has The Potential to Slow Disease Progression in DMD Patients Amenable to Exon 44 Skipping

#dmd #duchenne #exon44 #brogidirsen

👉 Read More: DMDWarrioR.com

5 months ago 0 0 0 0
Avidity Biosciences Announces Positive Pre-BLA Meeting with U.S. FDA for del-zota in DMD44 with a Submission Planned for Q1 2026

Avidity Biosciences Announces Positive Pre-BLA Meeting with U.S. FDA for del-zota in DMD44 with a Submission Planned for Q1 2026

Avidity Biosciences Announces Positive Pre-BLA Meeting with U.S. FDA for del-zota in DMD44 with a Submission Planned for Q1 2026

#dmd #duchenne #delzota #exon44 #exon44skipping #clinicaltrials #aviditybiosciences

👉 Read More: DMDWarrioR.com

5 months ago 0 0 0 0
Does Pulsed Electromagnetic Field (PEMF) Therapy Treat Duchenne Muscular Dystrophy and Initiate Dystrophin Production? In countries like Uzbekistan, information is being shared about the potential treatment of Duchenne Muscular Dystrophy with PEMF therapy. Pulsed Electromagnetic Field (PEMF) therapy is not a cure for Duchenne Muscular Dystrophy. Be careful not to let scammers take your money.

Does Pulsed Electromagnetic Field (PEMF) Therapy Treat Duchenne Muscular Dystrophy and Initiate Dystrophin Production? In countries like Uzbekistan, information is being shared about the potential treatment of Duchenne Muscular Dystrophy with PEMF therapy. Pulsed Electromagnetic Field (PEMF) therapy is not a cure for Duchenne Muscular Dystrophy. Be careful not to let scammers take your money.

Does Pulsed Electromagnetic Field (PEMF) Therapy Treat Duchenne Muscular Dystrophy and Initiate Dystrophin Production?

#pemf #dmd #duchenne

👉 Read More: DMDWarrioR.com

5 months ago 0 0 0 0
Tevard Biosciences Presents Data Demonstrating Production of Full-Length Protein with tRNA-Based Therapy for Duchenne Muscular Dystrophy Tevard Biosciences announced the presentation of new preclinical data showing potent restoration of full-length functional proteins in models of Duchenne muscular dystrophy (DMD) and dilated cardiomyopathy (DCM-TTNtv).

Tevard Biosciences Presents Data Demonstrating Production of Full-Length Protein with tRNA-Based Therapy for Duchenne Muscular Dystrophy Tevard Biosciences announced the presentation of new preclinical data showing potent restoration of full-length functional proteins in models of Duchenne muscular dystrophy (DMD) and dilated cardiomyopathy (DCM-TTNtv).

Tevard Biosciences Presents Data Demonstrating Production of Full-Length Protein with tRNA-Based Therapy for Duchenne Muscular Dystrophy

#dmd #duchenne

6 months ago 1 0 0 0
Cures of Duchenne (Detailed List of All Clinical Trials) List of all researches such as gene therapy, exon skipping, reducing inflammation and improving muscle growth & protection cures for duchenne muscular dystrophy.

Cures of Duchenne (Detailed List of All Clinical Trials) List of all researches such as gene therapy, exon skipping, reducing inflammation and improving muscle growth & protection cures for duchenne muscular dystrophy.

Cures of #Duchenne (Detailed List of All Clinical Trials for #DMD)

List of all researches such as #gene therapy, exon skipping, reducing inflammation and improving #muscle growth & protection cures for duchenne #muscular dystrophy.

dmdwarrior.com/dmd-treatmen...

6 months ago 1 0 0 0
Del-zota (Exon 44 Skipping Therapy) Demonstrated Reversal of Disease Progression Across Key Functional Endpoints in Phase 1/2 Trial With del-zota, normal dystrophin increased by 25% and creatine kinase decreased by 80%. Among exon 44 skipping therapies, the highest values ​​currently received have caused excitement. Unprecedented rapid reduction in creatine kinase (CK) to near normal levels maintained over 16 months of follow-up and 25% increase of normal in dystrophin production, reflecting sustained muscle fiber protection.

Del-zota (Exon 44 Skipping Therapy) Demonstrated Reversal of Disease Progression Across Key Functional Endpoints in Phase 1/2 Trial With del-zota, normal dystrophin increased by 25% and creatine kinase decreased by 80%. Among exon 44 skipping therapies, the highest values ​​currently received have caused excitement. Unprecedented rapid reduction in creatine kinase (CK) to near normal levels maintained over 16 months of follow-up and 25% increase of normal in dystrophin production, reflecting sustained muscle fiber protection.

Del-zota (Exon 44 Skipping Therapy) Demonstrated Reversal of Disease Progression Across Key Functional Endpoints in Phase 1/2 Trial

#dmd #duchenne #delzota #exon44 #exon44skipping #clinicaltrials #aviditybiosciences

👉 Read More: DMDWarrioR.com

6 months ago 2 0 0 0
Belief BioMed Announced Successful Completion of First Dose of BBM-D101 Gene Therapy Belief BioMed, an innovative biotechnology company focused on developing cutting-edge gene therapies, today announced that the registration clinical trial (CTR20252461) of its independently developed and produced BBM-D101 injection for Duchenne Muscular Dystrophy (DMD) has successfully completed the first patient dosing in Beijing, China.

Belief BioMed Announced Successful Completion of First Dose of BBM-D101 Gene Therapy Belief BioMed, an innovative biotechnology company focused on developing cutting-edge gene therapies, today announced that the registration clinical trial (CTR20252461) of its independently developed and produced BBM-D101 injection for Duchenne Muscular Dystrophy (DMD) has successfully completed the first patient dosing in Beijing, China.

Belief BioMed Announced Successful Completion of First Dose of BBM-D101 Gene Therapy

#dmd #duchenne #BBMD101 #BeliefBioMed #clinicaltrials

👉 Read More: DMDWarrioR.com

6 months ago 1 0 0 0
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Solid Biosciences Initiates Phase 3 Clinical Trial of SGT-003 Gene Therapy (IMPACT DUCHENNE) in Ambulant Males The phase 3 study (NCT07160634) of the SGT-003 gene therapy developed by Solid Biosciences is expected to begin in October 2025 and be completed in January 2029. The SGT-003 Phase 3 study will include 80 ambulant males, moreover the study locations have not yet been shared.

Solid Biosciences Initiates Phase 3 Clinical Trial of SGT-003 Gene Therapy (IMPACT DUCHENNE) in Ambulant Males The phase 3 study (NCT07160634) of the SGT-003 gene therapy developed by Solid Biosciences is expected to begin in October 2025 and be completed in January 2029. The SGT-003 Phase 3 study will include 80 ambulant males, moreover the study locations have not yet been shared.

Solid Biosciences Initiates Phase 3 Clinical Trial of SGT-003 Gene Therapy (IMPACT DUCHENNE) in Ambulant Males

#dmd #duchenne #sgt003 #genetherapy #clinicaltrials

👉 Read More: DMDWarrioR.com

6 months ago 0 0 0 0
Keros Therapeutics Presents Additional Clinical Data from its KER-065 Program at the American Society of Bone and Mineral Research 2025 Annual Meeting Phase 1 Trial in Healthy Participants of KER-065, a Modified Activin Receptor Ligand Trap, Supports Development in Duchenne Muscular Dystrophy and Bone Disorders.

Keros Therapeutics Presents Additional Clinical Data from its KER-065 Program at the American Society of Bone and Mineral Research 2025 Annual Meeting Phase 1 Trial in Healthy Participants of KER-065, a Modified Activin Receptor Ligand Trap, Supports Development in Duchenne Muscular Dystrophy and Bone Disorders.

Keros Therapeutics Presents Additional Clinical Data from its KER-065 Program at the American Society of Bone and Mineral Research 2025 Annual Meeting

#dmd #duchenne #ker065

👉 Read More: DMDWarrioR.com

6 months ago 1 0 0 0
GNR-097: Duchenne Gene Therapy Phase 1/2 Study Started by Generium Generium is a Russian innovative biotechnology company has received approval to conduct a clinical trial of GNR-097 gene therapy drug for the treatment of progressive Duchenne muscular dystrophy (DMD). The multicenter, single-blind, randomized placebo-controlled trial involves a single intravenous administration of the gene therapy drug to pediatric patients with progressive Duchenne muscular dystrophy. This Phase 1/2 study aims to evaluate the tolerability, safety, and efficacy of the drug GNR-097. #dmd #duchenne #gnr097 #genetherapy #clinicaltrials 👉 Read More: DMDWarrioR.com

GNR-097: Duchenne Gene Therapy Phase 1/2 Study Started by Generium Generium is a Russian innovative biotechnology company has received approval to conduct a clinical trial of GNR-097 gene therapy drug for the treatment of progressive Duchenne muscular dystrophy (DMD). The multicenter, single-blind, randomized placebo-controlled trial involves a single intravenous administration of the gene therapy drug to pediatric patients with progressive Duchenne muscular dystrophy. This Phase 1/2 study aims to evaluate the tolerability, safety, and efficacy of the drug GNR-097. #dmd #duchenne #gnr097 #genetherapy #clinicaltrials 👉 Read More: DMDWarrioR.com

GNR-097: Duchenne Gene Therapy Phase 1/2 Study Started by Generium

Generium is a Russian innovative biotechnology company has received approval to conduct a clinical trial of GNR-097 gene therapy drug for the treatment of progressive Duchenne muscular dystrophy (DMD).

👉 Read More: DMDWarrioR.com

6 months ago 1 0 0 0
🎈 SEPTEMBER 7, 2025 🎈 Support World Duchenne Awareness Day 2025 with DMDWarrior Theme of 2025: Make Cures Affordable for Duchenne We’re inviting YOU to stand with the #DuchenneCommunity and #DMDWarrior. Every post raises awareness. Every tag sparks a conversation. Every share brings us closer to a cure. 🚀 Be sure to tag @dmdwarrior and use these hashtags to amplify your impact: #WorldDuchenneAwarenessDay #dmdwarrior #SupportDuchenne #DMD #WDAD #UntilEveryoneHasACure #TogetherWeThrive #Becker #MuscularDystrophy #ExonSkipping 💪 Together We Are Stronger

🎈 SEPTEMBER 7, 2025 🎈 Support World Duchenne Awareness Day 2025 with DMDWarrior Theme of 2025: Make Cures Affordable for Duchenne We’re inviting YOU to stand with the #DuchenneCommunity and #DMDWarrior. Every post raises awareness. Every tag sparks a conversation. Every share brings us closer to a cure. 🚀 Be sure to tag @dmdwarrior and use these hashtags to amplify your impact: #WorldDuchenneAwarenessDay #dmdwarrior #SupportDuchenne #DMD #WDAD #UntilEveryoneHasACure #TogetherWeThrive #Becker #MuscularDystrophy #ExonSkipping 💪 Together We Are Stronger

🎈 SEPTEMBER 7, 2025 🎈 Support World Duchenne Awareness Day 2025 with DMDWarrior Theme of 2025: Make Cures Affordable for Duchenne We’re inviting YOU to stand with the #DuchenneCommunity and #DMDWarrior. Every post raises awareness. Every tag sparks a conversation. Every share brings us closer to a cure. 🚀 Be sure to tag @dmdwarrior and use these hashtags to amplify your impact: #WorldDuchenneAwarenessDay #dmdwarrior #SupportDuchenne #DMD #WDAD #UntilEveryoneHasACure #TogetherWeThrive #Becker #MuscularDystrophy #ExonSkipping 💪 Together We Are Stronger

🎈 SEPTEMBER 7, 2025 🎈 Support World Duchenne Awareness Day 2025 with DMDWarrior Theme of 2025: Make Cures Affordable for Duchenne We’re inviting YOU to stand with the #DuchenneCommunity and #DMDWarrior. Every post raises awareness. Every tag sparks a conversation. Every share brings us closer to a cure. 🚀 Be sure to tag @dmdwarrior and use these hashtags to amplify your impact: #WorldDuchenneAwarenessDay #dmdwarrior #SupportDuchenne #DMD #WDAD #UntilEveryoneHasACure #TogetherWeThrive #Becker #MuscularDystrophy #ExonSkipping 💪 Together We Are Stronger

🎈 SEPTEMBER 7, 2025 🎈 Support World Duchenne Awareness Day 2025 with DMDWarrior Theme of 2025: Make Cures Affordable for Duchenne We’re inviting YOU to stand with the #DuchenneCommunity and #DMDWarrior. Every post raises awareness. Every tag sparks a conversation. Every share brings us closer to a cure. 🚀 Be sure to tag @dmdwarrior and use these hashtags to amplify your impact: #WorldDuchenneAwarenessDay #dmdwarrior #SupportDuchenne #DMD #WDAD #UntilEveryoneHasACure #TogetherWeThrive #Becker #MuscularDystrophy #ExonSkipping 💪 Together We Are Stronger

🎈 SEPTEMBER 7, 2025 🎈 Support World Duchenne Awareness Day 2025 with DMDWarrior Theme of 2025: Make Cures Affordable for Duchenne We’re inviting YOU to stand with the #DuchenneCommunity and #DMDWarrior. Every post raises awareness. Every tag sparks a conversation. Every share brings us closer to a cure. 🚀 Be sure to tag @dmdwarrior and use these hashtags to amplify your impact: #WorldDuchenneAwarenessDay #dmdwarrior #SupportDuchenne #DMD #WDAD #UntilEveryoneHasACure #TogetherWeThrive #Becker #MuscularDystrophy #ExonSkipping 💪 Together We Are Stronger

🎈 SEPTEMBER 7, 2025 🎈

Support World Duchenne Awareness Day 2025 with DMDWarrior

Theme of 2025: Make Cures Affordable for Duchenne

#WorldDuchenneAwarenessDay #dmdwarrior #SupportDuchenne #DMD #WDAD #UntilEveryoneHasACure #TogetherWeThrive #Becker #MuscularDystrophy #ExonSkipping

6 months ago 2 0 0 0
How Does My Child Participate in Clinical Trials for Duchenne? Learn how your child can join Duchenne muscular dystrophy (DMD) clinical trials and discover trusted resources to find active studies near you. If your child has been diagnosed with Duchenne muscular dystrophy (DMD), you may be exploring every possible option for treatment and care—including clinical trials. But how does your child participate in a Duchenne clinical trial? And where can you find trials that are actively recruiting?

How Does My Child Participate in Clinical Trials for Duchenne? Learn how your child can join Duchenne muscular dystrophy (DMD) clinical trials and discover trusted resources to find active studies near you. If your child has been diagnosed with Duchenne muscular dystrophy (DMD), you may be exploring every possible option for treatment and care—including clinical trials. But how does your child participate in a Duchenne clinical trial? And where can you find trials that are actively recruiting?

How Does My Child Participate in Clinical Trials for Duchenne?

Learn how your child can join Duchenne muscular dystrophy (DMD) clinical trials and discover trusted resources to find active studies near you.

Learn More 👇

dmdwarrior.com/how-to-parti...

6 months ago 0 0 0 0
Support World Duchenne Awareness Day 2025 with DMDWarrior World Duchenne Awareness Day will be observed globally on September 7, 2025, with the theme “Make Cures Affordable for Duchenne.” This important day raises awareness about Duchenne Muscular Dystrophy (DMD), a rare genetic condition that causes progressive muscle weakness and affects thousands of individuals and families worldwide. Each year, the campaign calls for more attention, research, and action toward better treatments and, ultimately, a cure. Theme of 2025: Make Cures Affordable for Duchenne This year’s theme reflects the urgent and universal wish of the Duchenne community: access to effective and affordable cures for Duchenne Muscular Dystrophy (DMD). Families, patients, and advocacy groups are united in calling for equitable access to treatments, accelerated research, and global collaboration.

Support World Duchenne Awareness Day 2025 with DMDWarrior World Duchenne Awareness Day will be observed globally on September 7, 2025, with the theme “Make Cures Affordable for Duchenne.” This important day raises awareness about Duchenne Muscular Dystrophy (DMD), a rare genetic condition that causes progressive muscle weakness and affects thousands of individuals and families worldwide. Each year, the campaign calls for more attention, research, and action toward better treatments and, ultimately, a cure. Theme of 2025: Make Cures Affordable for Duchenne This year’s theme reflects the urgent and universal wish of the Duchenne community: access to effective and affordable cures for Duchenne Muscular Dystrophy (DMD). Families, patients, and advocacy groups are united in calling for equitable access to treatments, accelerated research, and global collaboration.

🎈 SEPTEMBER 7, 2025 🎈

Support World Duchenne Awareness Day 2025 with DMDWarrior

Theme of 2025: Make Cures Affordable for Duchenne

#WorldDuchenneAwarenessDay #dmdwarrior #SupportDuchenne #DMD #WDAD
#UntilEveryoneHasACure #TogetherWeThrive #Becker #MuscularDystrophy #ExonSkipping

7 months ago 2 1 0 0
ITF Therapeutics Announces Publication of Positive Long-Term Data Reinforcing Duvyzat (Givinostat) Efficacy and Safety as a Treatment for Duchenne Muscular Dystrophy Positive long-term safety and efficacy data for Duvyzat (Givinostat) as a treatment for Duchenne muscular dystrophy (DMD) were published today by ITF Therapeutics, an Italfarmaco affiliate. The data came from the company's open-label extensions of its Phase 2 and Phase 3 (EPIDYS) trials. 🎈 New data published in Annals of Clinical and Translational Neurology show that long-term treatment with givinostat can significantly delay loss of key mobility functions in patients with Duchenne muscular dystrophy. 🎈 Meaningful clinical benefit was observed across treatment groups, regardless of disease stage at initiation. 🎈 Givinostat remained well-tolerated during extended use, consistent with previous clinical trials. 🎈 Average follow-up in the extension study exceeded 36 treatment months, with some patients receiving treatment for over eight years since initiating givinostat treatment. #givinostat #duvyzat 👉 Read More: DMDWarrioR.com

ITF Therapeutics Announces Publication of Positive Long-Term Data Reinforcing Duvyzat (Givinostat) Efficacy and Safety as a Treatment for Duchenne Muscular Dystrophy Positive long-term safety and efficacy data for Duvyzat (Givinostat) as a treatment for Duchenne muscular dystrophy (DMD) were published today by ITF Therapeutics, an Italfarmaco affiliate. The data came from the company's open-label extensions of its Phase 2 and Phase 3 (EPIDYS) trials. 🎈 New data published in Annals of Clinical and Translational Neurology show that long-term treatment with givinostat can significantly delay loss of key mobility functions in patients with Duchenne muscular dystrophy. 🎈 Meaningful clinical benefit was observed across treatment groups, regardless of disease stage at initiation. 🎈 Givinostat remained well-tolerated during extended use, consistent with previous clinical trials. 🎈 Average follow-up in the extension study exceeded 36 treatment months, with some patients receiving treatment for over eight years since initiating givinostat treatment. #givinostat #duvyzat 👉 Read More: DMDWarrioR.com

ITF Therapeutics Announces Publication of Positive Long-Term Data Reinforcing Duvyzat (Givinostat) Efficacy and Safety as a Treatment for Duchenne Muscular Dystrophy

#givinostat #duvyzat

👉 Read More: DMDWarrioR.com

7 months ago 0 0 0 0
Agamree to be sold in United Arab Emirates, Saudi Arabia, Kuwait, Oman and Bahrain starting in 2026 Santhera Pharmaceuticals announces the signing of an exclusive agreement with Uniphar to manage the distribution of AGAMREE (vamorolone) in the United Arab Emirates, the Kingdom of Saudi Arabia, Kuwait, Oman and Bahrain, for the treatment of Duchenne muscular dystrophy (DMD) in patients four years of age and older.

Agamree to be sold in United Arab Emirates, Saudi Arabia, Kuwait, Oman and Bahrain starting in 2026 Santhera Pharmaceuticals announces the signing of an exclusive agreement with Uniphar to manage the distribution of AGAMREE (vamorolone) in the United Arab Emirates, the Kingdom of Saudi Arabia, Kuwait, Oman and Bahrain, for the treatment of Duchenne muscular dystrophy (DMD) in patients four years of age and older.

Agamree to be sold in United Arab Emirates, Saudi Arabia, Kuwait, Oman and Bahrain starting in 2026

#agamree #vamorolone #uae #bahrain #oman #saudi_arabia #kuwait

👉 Read More: DMDWarrioR.com

7 months ago 1 0 0 0
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FDA: ‘Elevidys Gene Therapy Will Continue to Be Used in Ambulatory DMD Patients’ In a statement Monday, it said the FDA's swift action eliminated a problem with Sarepta, allowing Elevidys to return to the market without another safety study as had been feared.

FDA: ‘Elevidys Gene Therapy Will Continue to Be Used in Ambulatory DMD Patients’ In a statement Monday, it said the FDA's swift action eliminated a problem with Sarepta, allowing Elevidys to return to the market without another safety study as had been feared.

FDA: ‘Elevidys Gene Therapy Will Continue to Be Used in Ambulatory DMD Patients’

#dmd #sarepta #roche #elevidys #fda #duchenne

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8 months ago 0 0 0 0
FDA Investigating Death of 8-Year-Old Brazilian Boy Who Received Elevidys The U.S. Food and Drug Administration is investigating the death of an 8-year-old boy who received Elevidys, a Sarepta Therapeutics gene therapy for Duchenne muscular dystrophy. The death occurred on June 7, 2025. The FDA has requested and voluntary suspension of product distribution as it investigates the safety concerns. The U.S. Food and Drug Administration (FDA) issued a press release announcing an investigation into the death of an eight-year-old Duchenne muscular dystrophy (Duchenne) patient who had received ELEVIDYS (delandistrogene moxeparvovec) gene therapy.

FDA Investigating Death of 8-Year-Old Brazilian Boy Who Received Elevidys The U.S. Food and Drug Administration is investigating the death of an 8-year-old boy who received Elevidys, a Sarepta Therapeutics gene therapy for Duchenne muscular dystrophy. The death occurred on June 7, 2025. The FDA has requested and voluntary suspension of product distribution as it investigates the safety concerns. The U.S. Food and Drug Administration (FDA) issued a press release announcing an investigation into the death of an eight-year-old Duchenne muscular dystrophy (Duchenne) patient who had received ELEVIDYS (delandistrogene moxeparvovec) gene therapy.

FDA Investigating Death of 8-Year-Old Brazilian Boy Who Received Elevidys

#dmd #sarepta #roche #elevidys #fda #duchenne

👉 Read More: DMDWarrioR.com

8 months ago 0 0 0 0
The European Medicines Agency (EMA) Issued a Negative Opinion on Elevidys A European Medicines Agency committee ruled that the therapy, called Elevidys, failed to show in studies that it improved patients’ movement abilities. Roche announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative opinion on the conditional marketing authorisation (CMA) for Elevidys (delandistrogene moxeparvovec) for ambulatory individuals aged three to seven years with Duchenne muscular dystrophy (DMD).

The European Medicines Agency (EMA) Issued a Negative Opinion on Elevidys A European Medicines Agency committee ruled that the therapy, called Elevidys, failed to show in studies that it improved patients’ movement abilities. Roche announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative opinion on the conditional marketing authorisation (CMA) for Elevidys (delandistrogene moxeparvovec) for ambulatory individuals aged three to seven years with Duchenne muscular dystrophy (DMD).

The European Medicines Agency (EMA) Issued a Negative Opinion on Elevidys

#dmd #sarepta #roche #elevidys #europe #ema

👉 Read More: DMDWarrioR.com

8 months ago 1 0 0 0
Roche Suspends Shipments of Elevidys Gene Therapy Outside of the United States

Roche Suspends Shipments of Elevidys Gene Therapy Outside of the United States

Roche Suspends Shipments of Elevidys Gene Therapy Outside of the United States

#dmd #sarepta #roche #elevidys

👉 Read More: DMDWarrioR.com

8 months ago 1 0 0 0