UAB is participating in a Phase I/II trial of AMT 191, a one time gene therapy designed to enable patients with classic Fabry disease to produce their own missing enzyme, potentially reducing the need ...
Personalized therapies for diabetes, while showing promise in international studies, is met with healthy skepticism from ...
Bayer has discontinued an early-stage clinical gene therapy for a rare genetic disorder in favor of a similar candidate. | ...
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SRPT stock up as enrollment begins in DMD gene therapy safety study
Shares of Sarepta Therapeutics SRPT rose about 2% yesterday after it announced that screening and enrollment are underway in ...
Enh3ance trial results indicate DTX301 reduces ammonia levels by 18% and supports dietary liberalization in patients with OTC deficiency.
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced ...
Days after FDA Commissioner Marty Makary appeared to malign uniQure’s AMT-130 in an interview with CNBC, the agency confirmed ...
Successful program buildout prioritizes defined patient flow, infusion access, cold-chain capabilities, and accountability ...
March 11 (Reuters) - Regenxbio said on Wednesday that interim data from a early-to-mid stage study of its experimental gene ...
Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...
NEW YORK and SAN SEBASTIÁN, Spain, March 17, 2026 /PRNewswire/ -- Apertura Gene Therapy, a biotechnology company developing ...
The Advanced Research Projects Agency for Health ( ARPA-H) awarded BioCurie up to $9.3 million to accelerate the development of scalable, data-driven genomic medicine production.
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