China's scientist scorned for baby gene editing is out of jail and back in the lab
He Jiankui, who shocked the world in 2018 by announcing the creation of the first gene-edited babies, tells NPR he's now working on a cure for Duchenne muscular dystrophy.(Image credit: Aowen Cao/NPR) (Source: NPR Health and Science)
Source: NPR Health and Science - June 8, 2023 Category: Consumer Health News Authors: John Ruwitch Source Type: news
His baby gene editing shocked ethicists. Now he's in the lab again
He Jiankui, who shocked the world in 2018 by announcing the creation of the first gene-edited babies, tells NPR he's now working on a cure for Duchenne muscular dystrophy.(Image credit: Aowen Cao/NPR) (Source: NPR Health and Science)
Source: NPR Health and Science - June 8, 2023 Category: Consumer Health News Authors: John Ruwitch Source Type: news
FibroGen ’s antibody fails PhIII in Duchenne muscular dystrophy
FibroGen’s top antibody candidate has failed a Phase III study in Duchenne muscular dystrophy, marking a disappointing start to a string of readouts slated for the next few months. Dubbed pamrevlumab, the drug is listed as a lead candidate for FibroGen alongside…#dubbedpamrevlumab #unlock (Source: Reuters: Health)
Source: Reuters: Health - June 7, 2023 Category: Consumer Health News Source Type: news
Researchers link death in gene-editing study to a virus used to deliver the treatment, not CRISPR
The lone volunteer in a gene-editing study targeting a rare form of Duchenne muscular dystrophy most likely died after having a reaction to the virus that delivered the therapy in his body (Source: ABC News: Health)
Source: ABC News: Health - May 31, 2023 Category: Consumer Health News Tags: Health Source Type: news
The Petri Dish: FDA delays Sarepta decision; Novartis buys Avrobio drug
The FDA has given itself a few more weeks to decide on the fate of Sarepta Therapeutics' latest treatment for Duchenne muscular dystrophy. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - May 25, 2023 Category: Pharmaceuticals Authors: Rowan Walrath Source Type: news
FDA delays decision on Sarepta ’s gene therapy for Duchenne muscular dystrophy
The Food and Drug Administration is delaying by one month a decision on the approval of a gene therapy for Duchenne muscular dystrophy, the treatment’s maker, Sarepta Therapeutics, said Wednesday. Sarepta said the FDA expects to complete the review of its gene therapy called SRP-9001 by June 22. A…#sareptatherapeutics #sarepta #fda (Source: Reuters: Health)
Source: Reuters: Health - May 25, 2023 Category: Consumer Health News Source Type: news
Should FDA Grant Accelerated Approval to Duchenne Muscular Dystrophy Gene Therapy?
(MedPage Today) -- On May 12, independent experts narrowly recommended that the FDA grant accelerated approval to Sarepta's new gene therapy, SRP-9001 (delandistrogene moxeparvovec), for the treatment of Duchenne muscular dystrophy (DMD). These... (Source: MedPage Today Neurology)
Source: MedPage Today Neurology - May 18, 2023 Category: Neurology Source Type: news
Roche ’s BTK inhibitor fenebrutinib significantly reduced brain lesions in people with relapsing forms of multiple sclerosis
Fenebrutinib is an investigational, potent and highly selective oralBruton ’s tyrosine kinase (BTK) inhibitor, the only reversible BTK inhibitor currently in Phase III multiple sclerosis (MS) trialsPhase II study met its primary and secondary endpoints by reducing the total number of new gadolinium-enhancing T1 brain lesions and significantly reducing the total number of new or enlarging T2 brain lesions compared to placeboThe safety profile offenebrutinib was consistent with previous and ongoing clinical trials across more than 2,400 people to dateBasel, 17 May 2023 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today p...
Source: Roche Media News - May 17, 2023 Category: Pharmaceuticals Source Type: news
Roche ’s BTK inhibitor fenebrutinib significantly reduced brain lesions in people with relapsing forms of multiple sclerosis
Fenebrutinib is an investigational, potent and highly selective oralBruton ’s tyrosine kinase (BTK) inhibitor, the only reversible BTK inhibitor currently in Phase III multiple sclerosis (MS) trialsPhase II study met its primary and secondary endpoints by reducing the total number of new gadolinium-enhancing T1 brain lesions and significantly reducing the total number of new or enlarging T2 brain lesions compared to placeboThe safety profile offenebrutinib was consistent with previous and ongoing clinical trials across more than 2,400 people to dateBasel, 17 May 2023 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today p...
Source: Roche Investor Update - May 17, 2023 Category: Pharmaceuticals Source Type: news
Duchenne Muscular Dystrophy Treatment Narrowly Captures Support of FDA Advisors
(MedPage Today) -- FDA advisors narrowly supported accelerated approval for Sarepta Therapeutics' investigational gene therapy for Duchenne muscular dystrophy (DMD) on Friday.
In a 8-to-6 vote, the agency's Cellular, Tissue, and Gene Therapies... (Source: MedPage Today Public Health)
Source: MedPage Today Public Health - May 14, 2023 Category: American Health Source Type: news
FDA advisers narrowly back accelerated approval of Sarepta gene therapy
U.S. Food and Drug Administration advisers voted 8-6 Friday to recommend the agency grant accelerated approval to Sarepta Therapeutics Inc.’s gene therapy for Duchenne muscular dystrophy despite questions about its clinical benefit and safety. Should it be approved, the first-of-its-kind potential…#dmd #fda #sarepta #sareptasrpt #bla (Source: Reuters: Health)
Source: Reuters: Health - May 13, 2023 Category: Consumer Health News Source Type: news
FDA advisers narrowly vote in favor of experimental gene therapy for rare muscle disease
SRP-9001, an experimental gene therapy that aims to slow or stop the progression of Duchenne muscular dystrophy, is being considered for accelerated approval by the FDA. (Source: CNN.com - Health)
Source: CNN.com - Health - May 13, 2023 Category: Consumer Health News Source Type: news
FDA advisers narrowly back first gene therapy for muscular dystrophy
In a split vote, advisers to the Food and Drug Administration recommended that the agency approve the first gene therapy for Duchenne muscular dystrophy, the most common form of the genetic illness. The vote, 8 to 6, came after a day of testimony from speakers for Sarepta Therapeutics, the maker…#sareptatherapeutics #fda #sarepta #craigmcdonald #nathanplasman #mikesinger #petermarks (Source: Reuters: Health)
Source: Reuters: Health - May 13, 2023 Category: Consumer Health News Source Type: news
FDA advisers narrowly back accelerated approval of Sarepta gene therapy
U.S. Food and Drug Administration advisers voted 8-6 Friday to recommend the agency grant accelerated approval to Sarepta Therapeutics Inc.’s gene therapy for Duchenne muscular dystrophy despite questions about its clinical benefit and safety. Should it be approved, the first-of-its-kind potential…#dmd #fda #sarepta #sareptasrpt #bla (Source: Reuters: Health)
Source: Reuters: Health - May 12, 2023 Category: Consumer Health News Source Type: news
Sarepta gets narrow 'yes' vote on Duchenne gene therapy in key FDA committee hearing
A committee of expert advisers to the U.S. Food and Drug Administration voted eight to six in favor of giving a Duchenne muscular dystrophy drug made by Sarepta Therapeutics Inc. accelerated approval in a key meeting. (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - May 12, 2023 Category: American Health Authors: Rowan Walrath Source Type: news
