Duchenne muscular dystrophy
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.
Sarepta Therapeutics has put in place several initiatives to help its gene therapy Elevidys return to growth, but recovery will take a long time, according to company executives.
As Q1 earnings arrive, three biotechs have big quarters ahead, with two—Amylyx and Neumora Therapeutics—betting at least partly on novel assets for obesity.
This year’s American Academy of Neurology meeting included a presentation that could one day set a new treatment standard for myasthenia gravis.
At Sarepta Therapeutics, we’ve seen it all. Here are the questions I believe we should be asking to move forward in Duchenne muscular dystrophy.
Biomarker data for Sarepta Therapeutics’ RNA programs, licensed from Arrowhead Pharmaceuticals, are “competitive” and “strong,” according to analysts at Jefferies, which projected over $1 billion in peak sales.
Sarepta Therapeutics says the FDA has agreed to review a regulatory package for Amondys 45 and Vyondys 53 after they failed a confirmatory trial, but whether the agency will agree to approve them is still unknown.
Dozens of biotechs reported earnings this week. BioSpace recaps key highlights from Capricor Therapeutics, Legend Biotech, Inovio and Allogene.
Solid Biosciences’ SGT-003 is the only late‑stage program to show early cardiac benefit across biomarkers and function, according to William Blair.
Capricor Therapeutics’ deramiocel was rejected in July 2025, potentially caught between Nicole Verdun, a former top biologics regulator at the FDA, and outgoing Vinay Prasad, director of the Center for Biologics Evaluation and Research.
PRESS RELEASES