Gene therapy for sickle cell disease shows strong clinician support but faces barriers in referral practices, knowledge, and ...
With promise to slow aging and cure some types of cancer, a new gene therapy presents cost and access hurdles for CT patients ...
In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later, the Pfizer ...
Biomarker data for Sarepta Therapeutics’ RNA programs, licensed from Arrowhead Pharmaceuticals, are “competitive” and “strong ...
An expert involved in the first successful gene therapy trial for Huntington's Disease discusses the trial results and ...
Researchers plan to begin the first patient trial in 2027 thanks to promising preclinical results from a gene therapy ...
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 ...
By Sneha S K and Kunal Das March 25 (Reuters) - The U.S. Food and Drug Administration has approved Denali Therapeutics' ...
Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.
The last available hemophilia gene therapy is temporarily unavailable, according to CSL Behring. | The company is working ...
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Ocugen falls as gene therapy for eye disease lags in phase II study
Shares of Ocugen OCGN declined 8.6% on Tuesday after the company announced 12-month data from the phase II ArMaDa study evaluating its novel modifier gene therapy, OCU410, for treating geographic ...
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