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 ...
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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 ...
Bayer has discontinued an early-stage clinical gene therapy for a rare genetic disorder in favor of a similar candidate. | ...
Mayo Clinic researchers used milk-based nanoparticles to deliver siRNA into bile duct cancer cells. This targeted therapy ...
Days after FDA Commissioner Marty Makary appeared to malign uniQure’s AMT-130 in an interview with CNBC, the agency confirmed ...
A pioneering gene therapy could help treat a rare seizure disorder called Dravet syndrome, according to new clinical trial ...
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 ...
Enh3ance trial results indicate DTX301 reduces ammonia levels by 18% and supports dietary liberalization in patients with OTC deficiency.
By Christy Santhosh March 2 (Reuters) - Intellia Therapeutics said on Monday the U.S. Food and Drug Administration has lifted the clinical hold on a late-stage trial of its gene therapy for a heart ...
NEW YORK and SAN SEBASTIÁN, Spain, March 17, 2026 /PRNewswire/ -- Apertura Gene Therapy, a biotechnology company developing ...
Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...
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