adrenal insufficiency in individuals with duchenne muscular dystrophy treated with glucocorticoids insights from the past current challenges and future directions

Original title: “Adrenal insufficiency in individuals with Duchenne muscular dystrophy treated with glucocorticoids: Insights from the past, current challenges, and future directions.”

safety and efficacy of aav based mini and micro dystrophin gene therapies in duchenne muscular dystrophy a systematic review and meta analysis of clinical trials

Original title: “Safety and efficacy of AAV-based mini- and micro-dystrophin gene therapies in Duchenne muscular dystrophy: a systematic review and meta-analysis of clinical trials.”

FDA extends its review of Capricor Therapeutics’ Duchenne muscular dystrophy cell therapy as an unhappy shareholder advocates for change; Merck and Moderna tout positive data for their mRNA-based cancer vaccine; Trump nominates White House aide Heidi Overton as FDA commissioner; and the oral obesity market heats up with rivals Eli Lilly and Novo Nordisk going head-to-head.

Original title: “Capricor’s DMD therapy still alive, Merck/Moderna’s cancer vax shines, FDA’s future revealed”

The FDA has granted a three-month extension for Capricor Therapeutics’ Duchenne muscular dystrophy hopeful deramiocel in order to review additional data and consider a refined indication. The new target action date for the therapy is November 22.

Original title: “Capricor’s Duchenne therapy has new life after FDA grants 3-month extension”

Capricor Therapeutics’ CEO Linda Marbán believes the FDA is willing to work with the biotech as the Aug. 22 deadline approaches for its Duchenne muscular dystrophy cell therapy deramiocel, with the biotech’s stock climbing 68% on the update.

Original title: “Capricor soars on potential FDA review extension, filing amendments”

antisense oligonucleotide selection scheme for rare duchenne muscular dystrophy mutations application to dmd exon 16 skipping

Original title: “Antisense oligonucleotide selection scheme for rare Duchenne muscular dystrophy mutations: Application to DMD exon 16 skipping.”

After an FDA advisory committee voted 9-3 on Wednesday against recommending Capricor Therapeutics’ Duchenne muscular dystrophy cardiomyopathy drug for approval, stakeholders allege an “ulterior motive” and “biased intentions.”

Original title: “Capricor CEO won’t rule out legal action against FDA after negative adcomm”

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”

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.”

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”

anxa11 suppression restores muscular function in the mdx mouse model of duchenne muscular dystrophy dmd

Original title: “ANXA11 suppression restores muscular function in the mdx mouse model of Duchenne muscular dystrophy (DMD).”