Thiard News@F4F

@newsen.bsky.social

1 month ago

Atossa Therapeutics Updates on Promising (Z)-Endoxifen Research at MDA Conference Atossa Therapeutics shared exciting updates on (Z)-endoxifen research at the MDA Clinical & Scientific Conference, showing promise for DMD treatments.

Atossa Therapeutics Updates on Promising (Z)-Endoxifen Research at MDA Conference #USA #Orlando,_FL #Duchenne_Muscular_Dystrophy #Atossa_Therapeutics #Z-Endoxifen

Thiard News@F4F

@newsen.bsky.social

1 month ago

Significant Progress in Duchenne Muscular Dystrophy Treatment: Brogidirsen's 4.5-Year Clinical Data Unveiled NS Pharma's presentation at the 2026 MDA Conference reveals impressive 4.5-year clinical trial results for Brogidirsen in treating DMD, showcasing long-term efficacy and safety.

Significant Progress in Duchenne Muscular Dystrophy Treatment: Brogidirsen's 4.5-Year Clinical Data Unveiled #USA #NS_Pharma #Paramus #Duchenne_Muscular_Dystrophy #Brogidirsen

Thiard News@F4F

@newsen.bsky.social

1 month ago

New Insights on Givinostat for Duchenne Muscular Dystrophy Highlighted at 2026 MDA Conference ITF Therapeutics has unveiled significant findings regarding Givinostat for Duchenne Muscular Dystrophy at the 2026 MDA Conference, focusing on safety and efficacy.

New Insights on Givinostat for Duchenne Muscular Dystrophy Highlighted at 2026 MDA Conference #United_States #ITF_Therapeutics #Concord #Duchenne_Muscular_Dystrophy #givinostat

Thiard News@F4F

@newsen.bsky.social

1 month ago

Avidity Biosciences to Present Groundbreaking Research at 2026 MDA Conference Avidity Biosciences is set to showcase innovative findings on RNA therapeutics at the 2026 MDA Conference, demonstrating its commitment to addressing muscular dystrophies.

Avidity Biosciences to Present Groundbreaking Research at 2026 MDA Conference #USA #Orlando #RNA_Therapeutics #Avidity_Biosciences #Duchenne_Muscular_Dystrophy

Thiard News@F4F

@newsen.bsky.social

2 months ago

PTC Therapeutics Takes Step Back on Translarna™ Regulatory Submission Following FDA Feedback PTC Therapeutics Inc. announces the withdrawal of the NDA for Translarna, aimed at treating Duchenne muscular dystrophy, following negative FDA feedback.

PTC Therapeutics Takes Step Back on Translarna™ Regulatory Submission Following FDA Feedback #USA #PTC_Therapeutics #Warren #Duchenne_Muscular_Dystrophy #Translarna

Thiard News@F4F

@newsen.bsky.social

2 months ago

Aucta Pharmaceuticals Announces Launch of PYQUVI Oral Suspension for DMD Aucta Pharmaceuticals prepares to release PYQUVI, a new oral suspension for treating Duchenne muscular dystrophy, offering patients a vital treatment option.

Aucta Pharmaceuticals Announces Launch of PYQUVI Oral Suspension for DMD #United_States #Basking_Ridge #Duchenne_Muscular_Dystrophy #Aucta_Pharmaceuticals #PYQUVI

Thiard News@F4F

@newsen.bsky.social

3 months ago

Atossa Therapeutics Achieves FDA Orphan Drug Designation for (Z)-Endoxifen for Duchenne Muscular Dystrophy Atossa Therapeutics has received Orphan Drug Designation from the FDA for its drug (Z)-Endoxifen, aimed at treating Duchenne muscular dystrophy, marking a significant advancement in their development efforts.

Atossa Therapeutics Achieves FDA Orphan Drug Designation for (Z)-Endoxifen for Duchenne Muscular Dystrophy #United_States #Seattle #Duchenne_Muscular_Dystrophy #Atossa_Therapeutics #(Z)-endoxifen

Thiard News@F4F

@newsen.bsky.social

4 months ago

Ractigen Therapeutics Marks Major Milestone with First Patient Dosed in DMD RAG-18 Study Ractigen Therapeutics celebrates an important milestone as it successfully doses the first patient in a trial for RAG-18, targeting Duchenne Muscular Dystrophy, a significant step towards innovative treatments.

Ractigen Therapeutics Marks Major Milestone with First Patient Dosed in DMD RAG-18 Study #China #Beijing #Duchenne_Muscular_Dystrophy #Ractigen #RAG-18

Thiard News@F4F

@newsen.bsky.social

4 months ago

Atossa Therapeutics Receives FDA Designation for (Z)-Endoxifen to Treat Duchenne Muscular Dystrophy Atossa Therapeutics gains a significant regulatory milestone as the FDA grants Rare Pediatric Disease designation for (Z)-Endoxifen targeting Duchenne Muscular Dystrophy, heralding new hope.

Atossa Therapeutics Receives FDA Designation for (Z)-Endoxifen to Treat Duchenne Muscular Dystrophy #USA #Seattle #Duchenne_Muscular_Dystrophy #Atossa_Therapeutics #Z-Endoxifen

Thiard News@F4F

@newsen.bsky.social

4 months ago

Upsher-Smith Unveils KYMBEE™ Tablets for Duchenne Muscular Dystrophy Treatment Upsher-Smith announces the launch of KYMBEE™ Tablets, designed for the treatment of Duchenne Muscular Dystrophy, supporting families with a Promise of Support.

Upsher-Smith Unveils KYMBEE™ Tablets for Duchenne Muscular Dystrophy Treatment #United_States #Upsher-Smith #Duchenne_Muscular_Dystrophy #Maple_Grove #KYMBEE

Thiard News@F4F

@newsen.bsky.social

5 months ago

Atossa Therapeutics Explores Potential of (Z)-Endoxifen for Duchenne Muscular Dystrophy Treatment Atossa Therapeutics is pursuing the use of (Z)-endoxifen for Duchenne Muscular Dystrophy, focusing on its effects and potential benefits for female carriers.

Atossa Therapeutics Explores Potential of (Z)-Endoxifen for Duchenne Muscular Dystrophy Treatment #United_States #Seattle #Duchenne_Muscular_Dystrophy #Atossa_Therapeutics #(Z)-endoxifen

Thiard News@F4F

@newsen.bsky.social

6 months ago

Duchenne Muscular Dystrophy Therapeutics Market Projected to Reach $6.6 Billion by 2033 Amidst Revolutionary Therapies The Duchenne Muscular Dystrophy therapeutics market is set to experience dramatic growth, moving from $2.19 billion in 2024 to $6.64 billion by 2033, thanks to gene and RNA therapies redefining treatment protocols.

Duchenne Muscular Dystrophy Therapeutics Market Projected to Reach $6.6 Billion by 2033 Amidst Revolutionary Therapies #None #gene_therapy #RNA_Therapies #Duchenne_Muscular_Dystrophy

Thiard News@F4F

@newsen.bsky.social

6 months ago

Brogidirsen Clinical Trial Results Offer Hope for DMD Treatment at 2025 World Muscle Society Congress NS Pharma presented promising 3.5-year clinical trial data for brogidirsen as a treatment for Duchenne muscular dystrophy at the 2025 World Muscle Society Congress.

Brogidirsen Clinical Trial Results Offer Hope for DMD Treatment at 2025 World Muscle Society Congress #None #NS_Pharma #Paramus #Duchenne_Muscular_Dystrophy #Brogidirsen

Thiard News@F4F

@newsen.bsky.social

6 months ago

FDA Approves Orphan Drug Status for NS-051/NCNP-04 to Combat Duchenne Muscular Dystrophy NS Pharma has received Orphan Drug Designation from the FDA for NS-051/NCNP-04, aimed at treating Duchenne muscular dystrophy through exon 51 skipping therapy.

FDA Approves Orphan Drug Status for NS-051/NCNP-04 to Combat Duchenne Muscular Dystrophy #United_States #NS_Pharma #Paramus #Duchenne_Muscular_Dystrophy #NS-051

Thiard News@F4F

@newsen.bsky.social

7 months ago

MLAB Biosciences Welcomes Dr. Edward M. Kaye as New Scientific Advisory Board Chair MLAB Biosciences has appointed Dr. Edward M. Kaye as Chair of its Scientific Advisory Board, aiming to enhance their neuromuscular therapy developments.

MLAB Biosciences Welcomes Dr. Edward M. Kaye as New Scientific Advisory Board Chair #United_States #Duchenne_Muscular_Dystrophy #Needham,_Massachusetts #MLAB_Biosciences #Edward_M._Kaye

Thiard News@F4F

@newsen.bsky.social

9 months ago

REGENXBIO's RGX-202 Gene Therapy Shows Promise for Duchenne Muscular Dystrophy REGENXBIO has unveiled promising preclinical results for RGX-202, a gene therapy aimed at treating Duchenne Muscular Dystrophy, highlighting functional benefits and muscular health.

REGENXBIO's RGX-202 Gene Therapy Shows Promise for Duchenne Muscular Dystrophy #United_States #Rockville #REGENXBIO #Duchenne_Muscular_Dystrophy #RGX-202

Thiard News@F4F

@newsen.bsky.social

10 months ago

Upsher-Smith Emphasizes Commitment to Duchenne Muscular Dystrophy at PPMD 2025 Conference Upsher-Smith demonstrates its dedication to the Duchenne muscular dystrophy community at the PPMD 2025 Annual Conference in Las Vegas.

Upsher-Smith Emphasizes Commitment to Duchenne Muscular Dystrophy at PPMD 2025 Conference #United_States #Las_Vegas #Upsher-Smith #Duchenne_Muscular_Dystrophy #Deflazacort_Tablets

Thiard News@F4F

@newsen.bsky.social

11 months ago

ITF Therapeutics Unveils Key Patient-Reported Data for DUVYZAT® in Duchenne Muscular Dystrophy at ISPOR 2025 ITF Therapeutics presents significant patient-reported outcome data for DUVYZAT® (givinostat) in Duchenne muscular dystrophy at ISPOR 2025, enhancing quality of life insights.

ITF Therapeutics Unveils Key Patient-Reported Data for DUVYZAT® in Duchenne Muscular Dystrophy at ISPOR 2025 #USA #DUVYZAT #Concord #Duchenne_Muscular_Dystrophy #Italfarmaco

Thiard News@F4F

@newsen.bsky.social

11 months ago

Investigation Announced by Pomerantz Law Firm for Sarepta Therapeutics Investors Pomerantz LLP is investigating potential securities fraud claims related to Sarepta Therapeutics, Inc., after alarming news led to a significant stock drop.

Investigation Announced by Pomerantz Law Firm for Sarepta Therapeutics Investors #United_States #New_York #Pomerantz_Law_Firm #Duchenne_Muscular_Dystrophy #Sarepta_Therapeutics

Thiard News@F4F

@newsen.bsky.social

1 year ago

Health Canada Accepts Priority Review for AGAMREE® to Treat Duchenne Muscular Dystrophy Kye Pharmaceuticals' AGAMREE® (vamorolone) is on the path to becoming Canada's first approved treatment for Duchenne muscular dystrophy, crucial for patients.

Health Canada Accepts Priority Review for AGAMREE® to Treat Duchenne Muscular Dystrophy #Canada #Mississauga #Duchenne_Muscular_Dystrophy #AGAMREE #Kye_Pharmaceuticals

Thiard News@F4F

@newsen.bsky.social

1 year ago

PTC Therapeutics: Regulatory Developments for Translarna in Europe Raise Concerns PTC Therapeutics announced the European Commission's decision not to renew Translarna’s marketing authorization, raising challenges for Duchenne muscular dystrophy treatments. Individual nations may still allow usage.

PTC Therapeutics: Regulatory Developments for Translarna in Europe Raise Concerns #United_States #PTC_Therapeutics #Warren,_NJ #Duchenne_Muscular_Dystrophy #Translarna

Thiard News@F4F

@newsen.bsky.social

1 year ago

Avidity Biosciences Reports Encouraging Results for Del-zota in Duchenne Muscular Dystrophy Phase 1/2 Trial Avidity Biosciences showcases promising topline data from its Phase 1/2 EXPLORE44 trial of del-zota, a potential treatment for Duchenne muscular dystrophy, demonstrating significant improvements.

Avidity Biosciences Reports Encouraging Results for Del-zota in Duchenne Muscular Dystrophy Phase 1/2 Trial #USA #San_Diego #Avidity_Biosciences #Duchenne_Muscular_Dystrophy #Del-zota

Thiard News@F4F

@newsen.bsky.social

1 year ago

NS Pharma Receives FDA Acceptance for Duchenne Muscular Dystrophy Treatment: A Hope for Families NS Pharma's acceptance from the FDA for its cell therapy deramiocel marks a significant advancement in treating Duchenne muscular dystrophy, offering hope to affected families.

NS Pharma Receives FDA Acceptance for Duchenne Muscular Dystrophy Treatment: A Hope for Families #USA #NS_Pharma #Paramus #Duchenne_Muscular_Dystrophy #deramiocel

Thiard News@F4F

@newsen.bsky.social

1 year ago

REGENXBIO to Present New Data for Duchenne Muscular Dystrophy at 2025 MDA Conference Discover the latest insights from REGENXBIO at the MDA Clinical Conference 2025. Key presentations on RGX-202's promising data for Duchenne Muscular Dystrophy.

REGENXBIO to Present New Data for Duchenne Muscular Dystrophy at 2025 MDA Conference #United_States #Dallas,_Texas #REGENXBIO #Duchenne_Muscular_Dystrophy #RGX-202

Thiard News@F4F

@newsen.bsky.social

1 year ago

GenAssist Ltd Achieves FDA Clearance for Innovative Base Editing Treatment for Duchenne Muscular Dystrophy GenAssist Ltd proudly announces FDA approval for its groundbreaking base editing drug GEN6050X, aimed at treating Duchenne Muscular Dystrophy, marking a significant advancement in gene therapy.

GenAssist Ltd Achieves FDA Clearance for Innovative Base Editing Treatment for Duchenne Muscular Dystrophy #China #Suzhou #Duchenne_Muscular_Dystrophy #GenAssist_Ltd #GEN6050X

Thiard News@F4F

@newsen.bsky.social

1 year ago

Cumberland Pharmaceuticals Achieves Landmark Success in DMD Heart Disease Trial with Ifetroban Cumberland Pharmaceuticals reports significant progress in treating Duchenne muscular dystrophy heart disease, marking an important milestone for DMD patients using ifetroban.

Cumberland Pharmaceuticals Achieves Landmark Success in DMD Heart Disease Trial with Ifetroban #USA #Nashville #Cumberland_Pharmaceuticals #Duchenne_Muscular_Dystrophy #ifetroban