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Muscular Dystrophy News1d
Precision’s gene-editing therapy gets FDA’s rare pediatric disease status
The FDA has granted rare pediatric disease designation to PBGENE-DMD, an experimental gene-editing therapy for Duchenne ...
Precision BioSciences Receives FDA Rare Pediatric Disease Designation for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy
Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® ...
InvestorsHub on MSN2d
Precision BioSciences Shares Surge Following FDA Rare Pediatric Disease Designation
Shares of Precision BioSciences, Inc. (NASDAQ:DTIL) jumped 17% on Wednesday after the company announced that its gene therapy ...
Nasdaq2d
Precision BioSciences Gets Rare Pediatric Disease Status For Duchenne Muscular Dystrophy Therapy - Nasdaq
(RTTNews) - Precision BioSciences, Inc. (DTIL) Wednesday said that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for its gene therapy candidate PBGENE ...
Asianet Newsable on MSN2d
Precision Bio's DMD Therapy Gets Rare Pediatric Status, But Stock Slides; Retail Crowd Sees ‘Good Risk-Reward’
The FDA designation could make Precision eligible for a valuable Priority Review Voucher, while preclinical data showed ...
STAT9dOpinion
Following second patient death, Duchenne muscular dystrophy families deserve answers about Elevidys
Duchenne muscular dystrophy families are sharing stories about Elevidys complications on social media. But they aren’t ...
No Accelerated FDA Approval For Edgewise's Rare Muscular Disease Drug; Shares Fall
Edgewise reports sustained functional stability in Becker patients and selects Phase 3 dose for Duchenne after positive Phase ...
Investing.com South Africa2d
Precision BioSciences stock jumps after FDA grants rare pediatric designation
Investing.com -- Precision BioSciences, Inc. (NASDAQ: DTIL) stock surged 17% on Wednesday after the company announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric ...
Contract Pharma2d
Precision BioSciences Gains FDA Rare Pediatric Disease Designation for PBGENE-DMD
Precision BioSciences, Inc. received a Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for PBGENE-DMD to treat Duchenne muscular dystrophy (DMD).
Second patient death reported with gene therapy for muscular dystrophy
Shares of Sarepta Therapeutics have plunged again after the drugmaker reported a second death in connection with its gene ...
Ticker: Second patient death reported with gene therapy for muscular dystrophy; Salmonella poisoning linked to pistachio cream
Ticker: Second patient death reported with gene therapy for muscular dystrophy; Salmonella poisoning linked to pistachio ...
The Victoria Advocate12d
UPSHER-SMITH SHOWCASES ITS COMMITMENT TO DUCHENNE MUSCULAR DYSTROPHY COMMUNITY AS A TRUSTED PARTNER IN PEDIATRIC RARE DISEASES AT THE PARENT PROJECT MUSCULAR DYSTROP…
MAPLE GROVE, Minn., June 16, 2025 /PRNewswire/ -- Upsher-Smith Laboratories, LLC (Upsher-Smith) today announced its participation at the Parent Project Muscular Dystrophy (PPMD) 2025 Annual ...