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REGENXBIO Announces Completion of Enrollment in Cohort 2 and Additional Positive Interim Data in AFFINITY DUCHENNE® Trial

February 7, 2024

REGENXBIO Announces Completion of Enrollment in Cohort 2 and Additional Positive Interim Data in AFFINITY DUCHENNE® Trial

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Sarepta Therapeutics Announces Positive Data for Duchenne Muscular Dystrophy Amenable to Skipping Exon 51

January 29, 2024

Sarepta Therapeutics reported positive data for SRP-5051, their next-generation exon skipping agent designed for individuals amenable to skipping Exon 51. In part B of the Phase 2 MOMENTURM study, ambulatory and […]

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Santhera Receives Approval for AGAMREE® (Vamorolone) as a Treatment for Duchenne Muscular Dystrophy in the United Kingdom

January 12, 2024

Santhera Receives Approval for AGAMREE® (Vamorolone) as a Treatment for DMD in the UK.

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PepGen Announces First Patient Dosed in CONNECT1-EDO51 Phase 2 Clinical Trial of PGN-EDO51 for Duchenne Muscular Dystrophy Patients Amenable to Exon 51 Skipping

January 8, 2024

PepGen Announces First Patient Dosed in CONNECT1-EDO51 Phase 2 Clinical Trial of PGN-EDO51 for Duchenne Muscular Dystrophy Patients Amenable to Exon 51 Skipping

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Dyne Therapeutics Announces Positive Initial Clinical Data from ACHIEVE Trial in DM1 Patients

January 3, 2024

Dyne Therapeutics Announces Positive Initial Clinical Data from ACHIEVE Trial in DM1 Patients and DELIVER Trial in DMD Patients

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Astellas study results are  available for ASP0367 (MA-0211) in Pediatric Male patients with Duchenne Muscular Dystrophy (0367-CL-0102)

November 30, 2023

Read Astellas community letter below:

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FDA clearance for DMD 

Solid Biosciences received clearance by the U.S. FDA for an Investigational New Drug (IND) application for SGT-003 for DMD 

November 14, 2023

Solid Biosciences Announces IND Clearance by FDA for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003 

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movie posters for a man in a wheelchair and his caregiver riding on a back, a boy wearing an astronaut helmet with his parent and a man in a wheelchair with a middle-aged man on the left and young woman on the right

Read and Watch These Four Stories About Disability This Holiday Season

November 2, 2023

The silver screen has been featuring more and more stories about people with disabilities, whether it be developmental, physical, or mental. With more representation in popular culture, comes more understanding […]

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Volunteering is Possible, Even with a Physical Disability

October 19, 2023

Volunteering within our community helps us lift everyone around us while making us feel good about our impact in the world. It’s something that is open to anyone, regardless of […]

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