Regenxbio Inc. RGNX shares are down on Wednesday after the company reported new interim data from its Phase 1/2 AFFINITY DUCHENNE trial of RGX-202. • Regenxbio stock is among today’s weakest ...
Solid Biosciences (SLDB) stock dips after new Phase 1/2 trial data for SGT-003, its gene therapy for Duchenne muscular dystrophy. Read more here, ...
Capricor Therapeutics’ deramiocel was rejected in July 2025, potentially caught between Nicole Verdun, a former top biologics regulator at the FDA, and Vinay Prasad, director of the Center for ...
Parent Project Muscular Dystrophy (PPMD), in partnership with the Foundation to Eradicate Duchenne (FED), today announced Representative Troy Balderson (OH-12), Senator Susan Collins (ME), Senator Amy ...
Interim observations from the Phase 2 TRAILHEAD study show continued improvement in handgrip strength, overall stability of elbow and shoulder ...
The selected dose in the pivotal phase is lower than for other drug candidates in clinical trials or gene therapy drugs for Duchenne.
March 11 (Reuters) - Regenxbio said on Wednesday that interim data from a early-to-mid stage study of its experimental gene therapy in patients with Duchenne muscular dystrophy showed continued ...
Dyne Therapeutics is plotting an approval application for z-rostudirsen in the back half of 2026—a push that will only be bolstered by the departure of controversial CBER chief Vinay Prasad, according ...
"Fast Track designation is an important regulatory milestone for PBGENE-DMD and reflects the significant unmet need in DMD," said Michael Amoroso, Chief Executive Officer of Precision BioSciences. "We ...
Durham-based Precision BioSciences, a gene-editing company, has received regulatory clearance to begin a clinical study of its potential treatment for Duchenne muscular dystrophy (DMD), a rare ...
Seven-year-old William, a Southern Oregon boy living with Duchenne muscular dystrophy, is going to get a never-before-tried surgery designed specifically for his genetic mutation. NBC5’s medical ...
While it’s established that Duchenne muscular dystrophy (DMD) causes a loss of muscle mass and strength, few studies have examined the gastrointestinal issues tied to DMD. Initial fractures are the ...
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