🎈 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

DMD Warrior - Gemeinschaft der Duchenne-Muskeldystrophie DMD Warrior wurde gegründet, um Kinder mit Duchenne-Muskeldystrophie zu unterstützen. Kinder mit DMD sind nicht allein.

Darf ich euch auf etwas aufmerksam machen?
#Duchenne #DMDWarrior
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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

Russia-Based Circle of Kindness Foundation Announces 18% Price Cut for Elevidys Gene Therapy As a result of the negotiations, the price of one of the most expensive drugs in the world was reduced by 18 percent, or 400 thousand euros, to 2.2 million euros," the press service of the Circle of Kindness Charitable State Foundation said. According to the Foundation’s press office, “as a result of the negotiations, the price of one of the most expensive drugs (Elevidys) in the world was reduced by 18%, that is by €400,000, to €2.2 million.” Take from the Rich, Give to the Poor Russia has set up a foundation called “Circle of Kindness,” which is financed by 2% of the individual income tax collected by high-income earners, despite the fact that rare (orphan) diseases pose a substantial financial burden on health systems and that access to these costly therapies is restricted. Since its founding in 2021, this sustainable mechanism has enabled the provision of medications for more than 24,000 children with rare diseases in 84 regions of the Russian Federation, totaling more than $2,4 billion USD. Resource allocation decisions are made using the process of health technology assessment. 2% of the high-income earners’ individual income taxes go toward funding the “Circle of Kindness” Foundation. Stated differently, each Russian citizen who earns more than 5 million rubles a year donates 2% of their taxes to the foundation. In addition to ensuring that children with rare and life-threatening illnesses have access to orphan medications, this dependable and sustainable financing method also removes the need for ongoing community fundraising initiatives. The “Circle of Kindness” has helped over 24,000 children in 84 locations since it was founded, spending 226,000,000 rubles ($2.4 billion USD equivalent) on this much-needed treatment.

Russia-Based Circle of Kindness Foundation Announces 18% Price Cut for Elevidys Gene Therapy

#dmd #elevidys #russia #duchenne #dmdwarrior

👉 Read More: DMDWarrioR.com

Duchenne Muscular Dystrophy (DMD) mRNA Studies May Provide Full-Length and Normal Dystrophin Production. Given that FDA-approved exon skipping therapies and gene therapy for Duchenne Muscular Dystrophy, which are known to not produce sufficient levels of dystrophin and thus do not completely eliminate Duchenne Muscular Dystrophy (DMD). Over the years, treatment options for DMD have been limited, but a promising area of research has emerged with the use of mRNA technology. These studies may enable production of full-length and normal dystrophin for DMD disease. Although exon skipping cures and gene therapy have recently been approved by the US Food and Drug Administration (FDA), none of these are thought to completely cure DMD, and there remains an urgent need to create methods that restore myofiber integrity, reverse the loss of muscle regenerative capacity, and address mitochondrial dysfunction, none of which are offered by current treatments. About 1 in 5,000 male newborns are affected by DMD, one of the most severe muscle degeneration illnesses. It is brought on by mutations in the X chromosome-related dystrophin gene. Young males with DMD experience gradual muscular degeneration, inflammation, fibrosis, and eventually pass away from heart and respiratory failure.

mRNA Studies in DMD Treatment: Future Therapeutic Candidates

Duchenne Muscular Dystrophy (DMD) mRNA Studies May Provide Full-Length and Normal Dystrophin Production.

#dmd #mrna #duchenne #dmdwarrior

👉 Read More: DMDWarrioR.com

We Call on Health Ministries and Pharmaceutical Manufacturers to Take Action on Duchenne Treatments Doesn't it upset you that children in your country are leaving this world because they can't access the necessary treatments? You are to blame. We call on all health ministries around the world and manufacturers of DMD treatments approved by the FDA and EMA to take action. It’s heart-wrenching to think that children in today’s world, in countries across the globe, are losing their lives far too early simply because they cannot access the medical treatments they so desperately need. This is the tragic reality for many children suffering from Duchenne Muscular Dystrophy (DMD), a devastating and rare genetic condition that leads to progressive muscle weakness and, ultimately, early death. The fact that life-saving treatments exist, yet these children are still dying due to a lack of access, is something that we cannot ignore. And the harsh truth is, we are all responsible for this crisis. Who is Guilty?

We Call on Health Ministries and Pharmaceutical Manufacturers to Take Action on Duchenne Treatments

Who is Guilty?

👉 Read More: dmdwarrior.com

#dmd #dmdwarrior #duchenne @fda.gov @ema.europa.eu @ec.europa.eu @who.int @anvisaoficial.bsky.social

Duchenne Muscular Dystrophy Market Grows: But Not All Families Have Access to Treatments While children with DMD are counting down the days until they regain their health, pharmaceutical companies are calculating their year-end revenues. This is what they call the Duchenne Muscular Dystrophy Market. If you have money, you can shop at this market. If you don't have money, your children are left alone to their fate. While DMD families struggle to restore their children to health, pharmaceutical companies whose primary mission is to heal children are focused on year-end profits. While research companies analyze the Duchenne Muscular Dystrophy market, pharmaceutical companies calculate their income. No one thinks about the children who will not have access to medicine.

Duchenne Muscular Dystrophy Market Grows: But Not All Families Have Access to Treatments

👉 Read More: dmdwarrior.com

#dmd #dmdwarrior #duchenne

The Challenge of Getting DMD Gene Therapy: Is Geography Destiny? Over the last decade, significant advancements have been made in the development of gene therapies for DMD, offering hope for a life-changing treatment. However, despite the promise these therapies hold, a major obstacle stands in the way of many patients: the prohibitively high cost of these treatments. This article explores the reasons behind the exorbitant price of Duchenne gene therapies and the devastating consequences for patients, families, and the healthcare system as a whole.

The Challenge of Getting DMD Gene Therapy: Is Geography Destiny?

#duchenne #dmd #duchennemusculardystrophy #dmdwarrior #duchenneawareness

👉 Read More: dmdwarrior.com

Satellos Announces Completed Enrollment of Phase 1 Clinical Trial of SAT-3247 in Healthy Volunteers SAT-3247 Announcement Summary 📌Company remains on track to report Phase 1a data from both the Single- and Multiple-Ascending Dose (SAD and MAD) cohorts in the healthy volunteer portion of the study in 1Q 2025 at an upcoming major medical meeting 📌The Phase 1b portion of the trial, in DMD patients, is underway with the intention of enrolling up to 10 adult volunteers with genetically confirmed DMD during 1Q 2025 📌Phase 2 IND filing on track to be submitted by end of 1Q 2025

Satellos Announces Completed Enrollment of Phase 1 Clinical Trial of SAT-3247 in Healthy Volunteers

#duchenne #dmd #duchennemusculardystrophy #dmdwarrior #duchenneawareness #satellos

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Entrada Therapeutics Receives UK Authorization to Begin ELEVATE-44-201 (Exon 44 Skipping) Entrada Therapeutics Receives UK Authorization to Begin ELEVATE-44-201 (Exon 44 Skipping Treatment), a Phase 1/2 Multiple Ascending Dose Clinical Study of ENTR-601-44 in Duchenne Muscular Dystrophy Patients. ELEVATE-44-201, a Phase 1/2 multiple ascending dose (MAD) clinical study of ENTR-601-44, was authorized by the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) and Research Ethics Committee for the Clinical Trial of an Investigational Medicinal Product. The study aims to treat Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation in the DMD gene that is amenable to exon 44 skipping.

Entrada Therapeutics Receives UK Authorization to Begin ELEVATE-44-201 (Exon 44 Skipping)

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#duchenne #dmd #duchennemusculardystrophy #dmdwarrior #duchenneawareness #uk #england #exon44skipping #entrada

Will Elevidys Receive Approval from the European Medicines Agency (EMA)? Patients with Duchenne muscular dystrophy (DMD) around Europe are anxiously awaiting the EMA's approval of Sarepta's application for its gene treatment Elevidys. Will Elevidys Receive Approval from the European Medicines Agency (EMA)? 📌 In Which Countries Is Elevidys Approved? 📌 In Which Countries Has Roche Applied for Authorization for Elevidys? 📌 Interim Results from Elevidys’ Embark Clinical Trial Could Impact Decision 📌 Why Are Sarepta and Roche Only Applying for Registration in Certain Countries? 📌 Families Concern if Sarepta for a Different Gene Therapy Will Not Be Approved by EMA 📌 Genethon and Sarepta Collaborated in 2017 📌 Pricing Policies Should Be Revised

Will Elevidys Receive Approval from the European Medicines Agency (EMA)?

👉 Read More: dmdwarrior.com

#dmd #duchenne #bmd #becker #genetherapy #dmdwarrior #dmdwarriors #duchennewarriors #duchennesmusculardystrophy #duchenneheroes #musculardystrophy #gene #dna #roche @roche #elevidys #sarepta

Roche Announces New Results from the EMBARK Study of Elevidys in Outpatients with Duchenne Muscular Dystrophy (DMD) Today, Roche revealed encouraging topline data from the second year of the EMBARK trial, a worldwide, double-blind, randomised phase III investigation of Elevidys (delandistrogene moxeparvovec), the first gene therapy to be authorized for the treatment of Duchenne muscular dystrophy. When compared to a predetermined propensity-weighted untreated external control group, statistically significant and clinically meaningful improvements were seen in three important motor function tests two years following Elevidys treatment: NSAA, TTR, and 10MWR. Between one and two years following dose, the functional differences between those receiving Elevidys and those in the external control group grew. When taken as a whole, these findings show that Elevidys has consistently benefited.

Roche Announces New Results from the EMBARK Study of Elevidys in Outpatients with Duchenne Muscular Dystrophy (DMD)

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#dmd #duchenne #bmd #becker #genetherapy #dmdwarrior #dmdwarriors #duchennesmusculardystrophy #gene #dna #brazil #roche @roche #elevidys #sarepta

dmd warrior dmdwarrior

WE WILL FIGHT DUCHENNE and WE WILL WIN 💪

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#dmd #duchenne #bmd #becker #genetherapy #dmdwarrior #dmdwarriors #duchennewarriors #duchennesmusculardystrophy #duchenne #duchenneawareness #duchenneheroes #musculardystrophy #gene #dna

Belief BioMed Announces IND Clearance by FDA for Duchenne Muscular Dystrophy Gene Therapy Candidate BBM-D101 The U.S. Food and Drug Administration (FDA) has approved an Investigational New Drug (IND) application for Belief BioMed’s (BBM) Duchenne Muscular Dystrophy (DMD) gene therapy candidate, BBM-D101. BBM is a forward-thinking biotechnology company that focuses on creating innovative gene therapies.

Belief BioMed Announces IND Clearance by FDA for Duchenne Muscular Dystrophy Gene Therapy Candidate BBM-D101

👉 Read More: dmdwarrior.com

#dmd #duchenne #bmd #becker #genetherapy #dmdwarrior #dmdwarriors #duchennewarriors #gene #dna #beliefbiomed #BBMD101 #china #shanghai

FDA Grants Rare Pediatric Disease Designation to Nippon Shinyaku’s Exon 51 Skipping Study (NS-051/NCNP-04) Nippon Shinyaku declared that NS-051/NCNP-04, a medication being developed to treat Duchenne Muscular Dystrophy (DMD), has been granted Rare Pediatric Disease Designation by the Food and Drug Administration (FDA). Nippon Shinyaku and the National Center of Neurology and Psychiatry jointly discovered the antisense oligonucleotide NS-051/NCNP-04. By omitting a portion of the dystrophin gene’s genetic material, NS-051/NCNP-04 creates a functioning dystrophin protein with a little shorter chain length, which should prevent the decline in muscle function.

FDA Grants Rare Pediatric Disease Designation to Nippon Shinyaku’s Exon 51 Skipping Study (NS-051/NCNP-04)

👉 Learn More: dmdwarrior.com

#dmd #duchenne #bmd #genetherapy #dmdwarrior #dmdwarriors #duchennewarriors #duchennesmusculardystrophy #exon51 #exon51skipping #nipponshinyaku #exonskipping

What is Bobcat mRNA? How Does Bobcat mRNA Work for Duchenne Muscular Dystrophy? What is Bobcat mRNA? Bobcat mRNA is a proprietary linear mRNA technology that produces high-quality mRNA encoding large proteins (greater than 13 kb) in a highly efficient and cost-effective manner. A lipid nanoparticle (LNP) delivers it. Elixirgen Therapeutics is first looking into how Bobcat mRNA can be used with dystrophin, which is the largest human gene known.

What is Bobcat mRNA? How Does Bobcat mRNA Work for Duchenne Muscular Dystrophy?

👉 Learn More: dmdwarrior.com

#dmd #duchenne #bmd #becker
#genetherapy #dmdwarrior #dmdwarriors #duchennewarriors #duchennesmusculardystrophy #duchenneheroes #musculardystrophy #gene #dna #bobcat #mrna #bobcatmrna

Insmed’s Gene Therapy (INS1201) Poised to Challenge Duchenne Muscular Dystrophy (DMD) For its INS1201 Duchenne (DMD) gene therapy, Insmed intends to initiate Phase I clinical trials in the first part of the year 2025. Insmed is getting ready to use its ground-breaking gene therapy, INS1201, to combat Duchenne muscular dystrophy. Insmed intends to start Phase I clinical trials for INS1201 in the first half of 2025 after the FDA approved its investigational new drug (IND) application in December 2024. What is Insmed INS1201 Gene Therapy? Learn More: dmdwarrior.com

Insmed’s Gene Therapy (INS1201) Poised to Challenge Duchenne Muscular Dystrophy (DMD)

👉 Learn More: dmdwarrior.com

#dmd #duchenne #bmd #becker #genetherapy #dmdwarrior #dmdwarriors #duchennewarriors #duchennesmusculardystrophy #duchenneheroes #musculardystrophy #gene #dna #insmed #ins1201

Solid Biosciences Releases Information on Clinical Trial for SGT-003 Gene Therapy Solid Biosciences shared detailed information about the clinical trials of its SGT-003 Gene Therapy developed for the treatment of Duchenne Muscular Dystrophy. Solid Biosciences shared that four patients have been dosed in the SGT-003 gene therapy clinical trial; it was well tolerated in all patients with no side effects observed; and data from the first three patients could be available in Q1 2025.

Solid Biosciences Releases Information on Clinical Trial for SGT-003 Gene Therapy

👉 Read More: dmdwarrior.com

#dmd #duchenne #bmd #becker #genetherapy #dmdwarrior #dmdwarriors #duchennewarriors #duchennesmusculardystrophy #duchenneawareness #gene #dna #solidbiosciences #sgt003

NICE Final Guidance for AGAMREE (Vamorolone) as a Treatment for Duchenne Muscular Dystrophy Is Positive for Santhera AGAMREE (vamorolone) has been recommended for use in the National Health Service (NHS) in England, Wales, and Northern Ireland for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 and up by the National Institute for Health and Care Excellence (NICE), according to Santhera Pharmaceuticals.

NICE Final Guidance for AGAMREE (Vamorolone) as a Treatment for Duchenne Muscular Dystrophy Is Positive for Santhera

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#dmd #duchenne #dmdwarrior #dmdwarriors #duchennesmusculardystrophy #agamree #vamorolone #santhera #NICE #uk #england #wales #northernireland

ImmunoForge Receives FDA IND Approval for Phase 2 Clinical Trial of ‘Pemziviptadil’ for DMD Cardiomyopathy The U.S. Food and Drug Administration (FDA) has approved ImmunoForge's Phase 2 clinical trial IND for "Pemziviptadil (development code name PF1804)," a treatment for DMD (Duchenne Muscular Dystrophy) cardiomyopathy. A medication for DMD cardiomyopathy, pemziviptadil is used once a week and is based on ImmunForge’s ELP Platform (Elastin Like Polypeptide Platform), a long-acting pharmacological platform. In order to improve cardiac function, pemziviptadil, a vasoactive intestinal peptide (VIP), preferentially works on the vasoactive intestinal peptide receptor 2 (VPAC2) to promote heart contraction and relaxation.

ImmunoForge Receives FDA IND Approval for Phase 2 Clinical Trial of ‘Pemziviptadil’ for DMD Cardiomyopathy

👉 Read More: dmdwarrior.com

#dmd #duchenne #bmd #becker #genetherapy #dmdwarrior #dmdwarriors #duchennesmusculardystrophy #musculardystrophy #gene #dna #ImmunoForge #Pemziviptadil #pf1804

Vamorolone (Agamree) Approved in Scotland The Scottish Medicines Consortium (SMC) approved the Agamree (Vamorolone) for use by NHS Scotland. The medication vamorolone, also marketed under the brand name Agamree, has been approved by the Scottish Medicines Consortium (SMC) as a therapy option for Duchenne muscular dystrophy in patients four years of age and older in Scotland as of today, Monday, January 13 2025.

Vamorolone (Agamree) Approved in Scotland

👉 Read More: dmdwarrior.com

#dmd #duchenne #bmd #genetherapy #dmdwarrior #dmdwarriors #duchennewarriors #duchennesmusculardystrophy #duchenneawareness #duchenneheroes #musculardystrophy #gene #dna #agamree #vamorolone #santhera #scotland #uk #england

Plans for DYNE-101 in DM1 and DYNE-251 in DMD Are Announced by Dyne Therapeutics Dyne Therapeutics announces clinical trial plans for DYNE-101 and DYNE-251, targeting myotonic dystrophy and Duchenne muscular dystrophy. Dyne Therapeutics announced positive clinical data for its therapies DYNE-101 and DYNE-251 during an investor event on January 10, 2025. DYNE-101 for myotonic dystrophy type 1 (DM1) demonstrated significant splicing correction and functional improvements, leading Dyne to plan a global Registrational Expansion Cohort of the ACHIEVE trial with a registrational dose of 6.8 mg/kg Q8W and a potential U.S. Accelerated Approval submission in H1 2026. Meanwhile, DYNE-251, targeting Duchenne muscular dystrophy (DMD), is also pursuing U.S. Accelerated Approval based on dystrophin as a surrogate endpoint, with data expected by late 2025 to support an early 2026 submission. Both programs show promise in transforming treatment paradigms for these genetic diseases.

Plans for DYNE-101 in DM1 and DYNE-251 in DMD Are Announced by Dyne Therapeutics

👉 Read More: dmdwarrior.com

#dmd #duchenne #bmd #becker #genetherapy #dmdwarrior #dmdwarriors #duchennewarriors #duchennesmusculardystrophy #duchenneheroes #musculardystrophy #gene #dna #dyne #dyne251 #dyne101

Nippon Shinyaku Releases First Clinical Trial (First in Human) of Brogidirsen NS-089/NCNP-02 for the Treatment of Duchenne Nippon Shinyaku Corporation has announced the first human trial results of its drug 'Brogidirsen' (NS-089/NCNP-02), which was developed for exon 44 skipping in the treatment of Duchenne muscular dystrophy (DMD). The National Center of Neurology and Psychiatry is announced the publication of a research paper in Cell Reports Medicine detailing the results of an investigator-initiated trial for NS-089/NCNP-02, known as “brogidirsen.” This innovative treatment for Duchenne muscular dystrophy (DMD), jointly developed with Nippon Shinyaku Co., Ltd., represents a significant advancement in exon 44 skipping therapy. What is Brogidirsen? Brogidirsen (NS-089/NCNP-02) is a nucleic acid drug co-discovered by Nippon Shinyaku and NCNP, and is expected to be a therapeutic drug for DMD patients with dystrophin gene mutations amenable to exon 44 skipping.

Nippon Shinyaku Releases First Clinical Trial (First in Human) of Brogidirsen NS-089/NCNP-02 for the Treatment of Duchenne

What is Brogidirsen?

👉 Read More: dmdwarrior.com

#dmd #duchenne #bmd #becker #genetherapy #dmdwarrior #dmdwarriors #nipponshinyaku #brogidirsen #NS089 #exonskipping #exon44

Avidity Biosciences Prepares for Three Rare Muscle Disease Programs’ 2025 BLA Submission and Commercialization Avidity Biosciences, Inc., a biopharmaceutical company producing Antibody Oligonucleotide Conjugates (AOC) RNA therapies, announced its progress and goals for a transformative 2025. A biopharmaceutical firm dedicated to developing a novel class of RNA treatments known as Antibody Oligonucleotide Conjugates (AOCsTM), Avidity Biosciences, Inc., today reported its advancements and plans for a game-changing year in 2025. These significant expected milestones include completing three potentially registrational programs, submitting a Biologics License Application (BLA) for delpacibart zotadirsen (del-zota) for individuals with Duchenne muscular dystrophy mutations amenable to exon 44 skipping (DMD44), getting ready for several product launches in DMD44, myotonic dystrophy type 1 (DM1), and facioscapulohumeral muscular dystrophy (FSHD), and establishing a completely integrated, international business to support an expanding AOC pipeline in precision cardiology and neuromuscular diseases.

Avidity Biosciences Prepares for Three Rare Muscle Disease Programs’ 2025 BLA Submission and Commercialization

👉 Read More: dmdwarrior.com

#dmd #duchenne #bmd #becker #genetherapy #dmdwarrior #dmdwarriors #duchennesmusculardystrophy #duchenne #gene #dna #avidity #aviditybiosciences #raredisease

Turkish DMD Patients Who are Eligible for Exon 51 Skipping Treatment Request Their Medications from the Ministry of Health 🇹🇷 According to the information sent to DMD Warrior by DMD Dayanisma Community, it was understood that Eteplirsen (Exondys 51) was included in the scope of payment by the Ministry of Health in the official response given to the written question submitted by Talih Ozcan, a member of parliament in Turkey, regarding the diagnosis, treatment and social rights of Duchenne Muscular Dystrophy (DMD) patients. According to the DMD Dayanisma Community, patients who are eligible for Exon 51 skipping treatment are not aware that they can get their medication from the Ministry of Health because official associations in Turkey have not made any announcements on their websites about this issue.

Turkish DMD Patients Who are Eligible for Exon 51 Skipping Treatment Request Their Medications from the Ministry of Health 🇹🇷

👉 Read More: dmdwarrior.com

#dmd #duchenne #bmd #becker #dmdwarrior #dmdwarriors #eteplirsen #ataluren #exondys51 #sarepta #turkish #turkey

Percheron Disappointed in Avicursen (ATL1102) Study For Duchenne (DMD) Percheron plans to broaden pipeline following Avicursen (ATL1102) disappointment. After disappointing Phase IIb results for avicursen last month, Percheron will adopt a three-pronged business strategy. Percheron Therapeutics has announced that it will investigate avicursen (ATL1102) in other indications besides Duchenne muscular dystrophy (DMD) following disappointing Phase IIb results last month. In a letter to shareholders published today (6 January), the chair of the board Dr Charmaine Gittleson, alongside non-executive director Dr Gil Price and managing director Dr James Garner, told shareholders that the company “will undertake a broad strategic review of the company’s pipeline to more comprehensively evaluate alternative sources of value within the current assets”. The review follows negative topline results from the Phase IIb trial (NCT05938023) of avicursen in boys with non-ambulatory DMD. Announced on 18 December, the results demonstrated that the trial did not meet its primary endpoint, which was the Performance of the Upper Limb 2.0 (PUL2.0) score at week 25 compared to placebo.

Percheron Disappointed in Avicursen (ATL1102) Study For Duchenne (DMD)

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#dmd #duchenne #bmd #becker #genetherapy #dmdwarrior #dmdwarriors #duchennesmusculardystrophy #duchenne #musculardystrophy #percheron #avicursen #atl1102 #NCT05938023 #percherontherapeutics

Santhera Enters into Supply and Distribution Agreement for AGAMREE (Vamorolone) with Clinigen Group "We look forward to working with Santhera to make AGAMREE available to patients globally on an unlicensed basis in those territories where it is not currently licensed or reimbursed. ” said Julie Gosper, Senior Vice President, Europe and Partner Markets at Clinigen. Santhera Pharmaceuticals announces the signing of an exclusive agreement with Clinigen Group to manage the supply and distribution of AGAMREE® (vamorolone) in countries where the product is not otherwise commercially available. Santhera Pharmaceuticals and Clinigen Group announce that they have signed a Supply and Distribution Agreement for AGAMREE in countries where the product can currently not be commercially obtained via Santhera or one of its current distribution partners. The agreement will enable access to AGAMREE on a case-by-case basis for patients with Duchenne muscular dystrophy (DMD) in situations where the treating physician deems there to be no suitable alternatives and where regulations allow for it. This agreement allows Santhera to receive the majority share of revenue generated from the supply of product in these additional countries.

Santhera Enters into Supply and Distribution Agreement for AGAMREE (Vamorolone) with Clinigen Group

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#dmd #duchenne #bmd #becker #dmdwarrior #dmdwarriors #duchennewarriors #duchennesmusculardystrophy #duchenne #agamree #vamorolone #santhera #Clinigen #clinigengroup

Potential Upcoming New Gene Therapies for Duchenne Muscular Dystrophy What other new gene therapy studies are there besides Elevidys? Let’s remember the new gene therapies that will be an alternative to Elevidys.

Potential Upcoming New Gene Therapies for Duchenne Muscular Dystrophy

What other new gene therapy studies are there besides Elevidys?

👉 Read More: dmdwarrior.com

#dmd #duchenne #bmd #becker #genetherapy #dmdwarrior #dmdwarriors #duchennewarriors #duchenne #newgenetherapies

One day our children will be able to enjoy life freely. dmd warrior. dmdwarrior.

One day our children will be able to enjoy life freely.

Together, we are stronger. 💪

#dmd #duchenne #bmd #becker #genetherapy #dmdwarrior #dmdwarriors #duchennewarriors #duchennesmusculardystrophy #duchenne #musculardystrophy #gene #dna #newyear #snowboarding #2025 #skiing #world #followme

Capricor Therapeutics Completes Submission of Biologics License Application to the U.S. FDA for Deramiocel for the Treatment of Duchenne Muscular Dystrophy -If approved, deramiocel would be first approved therapy for Duchenne muscular dystrophy cardiomyopathy. -BLA submission triggers $10 million milestone payment to Capricor from Nippon Shinyaku.

Capricor Therapeutics Completes Submission of Biologics License Application to the U.S. FDA for Deramiocel for the Treatment of Duchenne Muscular Dystrophy

#dmd #duchenne #bmd #becker #genetherapy #dmdwarrior #dmdwarriors #duchennewarriors #duchenne #musculardystrophy #Capricor #Deramiocel #fda