During a perplexing advisory committee meeting in which FDA reviewers focused largely on the Phase 3 HOPE-3 trial’s secondary endpoint of cardiac function, experts pointed to “very fragile” data. The committee voted 9-3 against approval of deramiocel.

Original title: “FDA reviewers vote against approval of Capricor’s DMD therapy in chaotic adcomm meeting”

During a perplexing advisory committee meeting in which FDA reviewers focused largely on the Phase 3 HOPE-3 trial’s secondary endpoint of cardiac function, experts pointed to “very fragile” data. The committee voted 9-3 against approval of deramiocel.

Original title: “FDA advisers vote against approval of Capricor’s DMD therapy in chaotic adcomm meeting”

At a contentious meeting with possible implications for other rare disease drugmakers, outside experts ultimately determined that Capricor didn't prove deramiocel's benefits in Duchenne-related cardiomyopathy.

Original title: “Capricor Duchenne cell therapy voted down by FDA panel”

The FDA's rebuff of Replimune comes a day after the agency similarly called into question the Phase 3 trial for Capricor’s Duchenne muscular dystrophy cell therapy.

Original title: “FDA says Replimune’s melanoma data package is ‘not interpretable,’ setting up tough adcomm”

In briefing documents published Monday ahead of an advisory committee meeting for deramiocel, the FDA disagreed with Capricor's assertion that a Phase 3 trial met its primary outcome measures.

Original title: “FDA disputes Capricor's claims of Phase 3 success for DMD cell therapy”

After leaving Tessera Therapeutics earlier this month, Michael Severino will take over Sarepta Therapeutics as the biotech awaits two key regulatory decisions in Duchenne muscular dystrophy.

Original title: “Sarepta names former AbbVie R&D exec Michael Severino as next CEO”

If approved, Dyne Therapeutics’ zeleciment rostudirsen could “capture the majority” of the exon-51 Duchenne muscular dystrophy market, given its better efficacy and dosing profile versus Sarepta Therapeutics’ exon-skipping therapy Exondys 51, the current standard of care, according to Oppenheimer.

Original title: “Dyne gets early 2027 decision date for Duchenne therapy, analysts expect ‘smooth’ review”

clinical pathological and genetic characteristics of 23 dmd patients in northern china

Original title: “Clinical, pathological, and genetic characteristics of 23 DMD patients in northern China.”

SEGRAM aims to offer an alternative to glucocorticoid‑based treatment

Original title: “Grünenthal receives FDA designations for tegacorat in Duchenne muscular dystrophy”

evaluating the impact of different natural history modeling methods on cost effectiveness decisions a case study in duchenne muscular dystrophy

Original title: “Evaluating the Impact of Different Natural History Modeling Methods on Cost-Effectiveness Decisions: A Case Study in Duchenne Muscular Dystrophy.”

prevalence and characteristics of cardiac arrhythmia in duchenne muscular dystrophy a retrospective single center holter electrocardiographic monitoring study

Original title: “Prevalence and Characteristics of Cardiac Arrhythmia in Duchenne Muscular Dystrophy: A Retrospective, Single-Center Holter Electrocardiographic Monitoring Study.”

Analysts homed in on Duchenne muscular dystrophy and myotonic dystrophy type 1 assets during first quarter earnings as major players like REGENXBIO and Novartis as well as Dyne, Wave, Solid and Sarepta near the regulatory finish line.

Original title: “REGENXBIO, Novartis, Dyne near FDA submissions in muscular dystrophies”

identification of biomarkers associated with pathological dynamic progression in dystrophinopathies

Original title: “Identification of biomarkers associated with pathological dynamic progression in dystrophinopathies.”

While the company claimed a victory, analysts said data from a Phase 1/2 study fell short of expectations. Entrada shares subsequently tumbled more than 50%.

Original title: “Entrada shares dive as Duchenne results disappoint”

Entrada Therapeutics saw a 2.36% dystrophin increase in patients treated with its oligonucleotide asset—a number that fell short of the company’s prior guidance of a double-digit improvement.

Original title: “Entrada crashes as Duchenne therapy comes in ‘below expectations’ in early study”