Duchenne muscular dystrophy
Edgewise Therapeutics will now focus on a handful of cardiovascular programs including EDG-7500 for hypertrophic cardiomyopathy thanks to the non-dilutive capital from France’s Servier.
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.
Shares of REGENXBIO declined 37% on a mixed data readout and other updates from the company’s first quarter earnings call Thursday.
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.
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