safety and efficacy of delpacibart zotadirsen in duchenne muscular dystrophy mutations amenable to exon 44 skipping in the usa explore44 a randomised placebo controlled double blind phase 1 2 trial

Original title: “Safety and efficacy of delpacibart zotadirsen in Duchenne muscular dystrophy mutations amenable to exon 44 skipping in the USA (EXPLORE44): a randomised, placebo-controlled, double-blind, phase 1/2 trial.”

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”

Kaos Capital wants a “governance reset” to alter the Duchenne cell therapy maker’s strategic course. Elsewhere, Bristol Myers furthered efforts to build an “AI-powered” discovery engine.

Original title: “Shareholder pressures Capricor; Lilly dives deeper into vaccines”

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”

The FDA is “willing to review” an updated submission for Capricor’s Duchenne cell therapy, its CEO said. Elsewhere, a heart drug battle spilled into court and Rigel priced the first “Protac” medicine.

Original title: “Capricor shares spike on CEO comments; Cytokinetics sues Bristol Myers”

The Biotech Consortium to Accelerate ... therapies for serious neurological and rare diseases, including amyotrophic lateral sclerosis, frontotemporal dementia, Angelman syndrome, and Duchenne muscular dystrophy. BCAI's mission is to keep American clinical trials in the U.S., ... The Biotech Consortium to Accelerate Innovation (BCAI) unites more than a dozen innovative biotechnology companies developing transformative therapies for serious neurological and rare diseases, including amyotrophic lateral sclerosis, frontotemporal dementia, Angelman syndrome, and Duchenne muscular dystrophy. BCAI's mission is to keep American clinical trials in the U.S., so American patients have the earliest possible access to U.S.-developed medical innovation. CAMBRIDGE, Mass.--(BUSINESS WIRE)--A new survey reveals that U.S. biotechnology companies focused on developing treatments for rare and serious diseases overwhelmingly prefer to conduct first-in-human (FIH) clinical trials in the United States. However, historical obstacles posed by the U.S. Through strategic cost-saving initiatives, robust business partnerships, educational and networking opportunities, and proactive advocacy, MassBio empowers its members to launch the next generation of medical advancements to deliver the cures and therapies that enhance patient lives. ... 1U.S. Food and Drug Administration. Estimating the Maximum Safe Starting Dose in Initial Clinical Trials for Therapeutics in Adult Healthy Volunteers: Guidance for Industry. Instead, they are asking the FDA to implement the 10x safety margin as outlined in the Agency’s own 2005 guidance, Estimating the Maximum Safe Starting Dose in Initial Clinical Trials1. Restore predictable review timelines that reduce costly holds and rework. Strengthen sponsor-reviewer communication so trial design is driven by science, not procedural ambiguity. Ensure better alignment of review criteria with disease severity and life expectancy.

Original title: “New Survey Finds U.S. Biotechs Advancing Treatments for Rare and Serious Diseases Want to Run Clinical Trials in U.S. but Regulatory Hurdles are Sending Them Abroad”

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”