Por: RTTNews Health May 24, 2023
Sarepta Therapeutics, Inc. () on Wednesday provided the following update on the Biologics License Application (BLA) for SRP-9001 (delandistrogene moxeparvovec), which is currently under review for the treatment of ambulant individuals with Duchenne muscular dystrophy (DMD) who have a confirmed mutation of the DMD gene.The company said the FDA has indicated that, subject to the completion of the BLA review, it is working toward potentially... + full article
The Boston Globe USA Business May 24, 2023
The Food and Drug Administration is delaying by one month its long-awaited decision on whether to approve a gene therapy for a deadly inherited muscle-wasting disease that afflicts more than 10,000 boys and young men nationwide, according to the Cambridge biotech that developed... + más
FDA advisers recommend accelerated approval of a Sarepta gene therapy | The Boston Globe
First gene therapy for deadly form of muscular dystrophy gets FDA approval for young kids | ABC News
The Boston Globe USA Business May 23, 2023
Regulators are to decide within days whether to clear a Cambridge biotech’s gene therapy for a deadly muscle-wasting disease that afflicts more than 10,000 boys and young men nationwide, even though questions remain about whether the treatment is safe and effective.The... + más
Gene therapy has made some recent progress—is it enough? | Ars Technica
The Boston Globe USA Business May 13, 2023
An panel of advisers Friday recommended that the Food and Drug Administration speed up approving a Duchenne muscular dystrophy treatment developed by Cambridge biotech Sarepta Therapeutics, despite questions about its clinical benefit and safety.By an 8 to 6 vote, the FDA... + más
Under pressure from patients, FDA faces tough choices on experimental gene therapy | The Boston Globe
FDA delays decision on Sarepta’s controversial gene therapy for muscular dystrophy | The Boston Globe
Fox News USA Sports February 16, 2023
Nyheim Hines had a challenging 2022 season as he was traded from the Colts to the after he spent the first four full seasons of his NFL career in Indianapolis. But for the veteran running back, life on the field can pale in comparison to what’s happening off it.Hines recently... + más
Bills' Nyheim Hines believes NFL will listen to players about field conditions, shares insight on Josh Allen | Fox News
AFC-best Bills trade Zack Moss, pick to Colts for RB Nyheim Hines | ABC News
The Boston Globe USA Business November 05, 2022
A man with muscular dystrophy who was first in line to receive an experimental gene editing therapy tailor-made to treat the cause of his rare form of the disease has died.The creation of for Terry Horgan, 27, was helmed by the Boston- and Connecticut-based nonprofit Cure Rare... + más
Boston startup Ascidian Therapeutics charts vision for therapies that ‘rewrite RNA’ | The Boston Globe
Death in CRISPR gene therapy study sparks search for answers | ABC News
Portland Press Herald USA Health November 05, 2022
The lone volunteer in a unique study involving a gene-editing technique has died, and those behind the trial are now trying to figure out what killed him. Terry Horgan, a 27-year-old who had Duchenne muscular dystrophy, died last month, according to Cure Rare Disease, a... + más
Death in CRISPR gene therapy study sparks search for answers | Associated Press
RTTNews USA Health September 29, 2022
Genetic medicine maker Sarepta Therapeutics, Inc. () announced Thursday that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for the accelerated approval of SRP-9001 (delandistrogene moxeparvovec) to treat ambulant patients... + más
Diageo Debuts Its 2022 Special Releases Collection | Forbes
Parents of son diagnosed with Duchenne muscular dystrophy are hopeful cure will be found soon | ABC7
About iurex | Privacy Policy | Disclaimer |