Por: RTTNews Health June 23, 2023
Sarepta Therapeutics, Inc. announced Friday that the U.S. Food and Drug Administration or FDA has accelerated approval of ELEVIDYS (delandistrogene moxeparvovec-rokl) for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.DMD is a rare and serious genetic condition which worsens over time, leading to weakness and wasting away of the body's... + full article
ABC News USA Health June 23, 2023
WASHINGTON -- The first gene therapy for a deadly form of muscular dystrophy received preliminary U.S. approval on Thursday despite concerns from some government scientists about the treatment's ability to help boys with the inherited disease.The Food and Drug... + más
Bills' Nyheim Hines opens up about family members' struggles with muscular dystrophy, hopes to inspire people | Fox News
First gene therapy for deadly form of muscular dystrophy gets FDA approval for young kids | WPLG Local 10
WPLG Local 10 USA Health June 23, 2023
WASHINGTON – The first gene therapy for a deadly form of muscular dystrophy received preliminary U.S. approval on Thursday despite concerns from some government scientists about the treatment's ability to help boys with the inherited disease.The Food and Drug... + más
First gene therapy for deadly form of muscular dystrophy gets FDA approval for young kids | ABC News
Time USA Health June 23, 2023
For genetic conditions like Duchenne muscular dystrophy, there is little doctors can do to slow or treat the condition other than trying to manage symptoms, since only addressing the genetic changes responsible can help halt the disease. Earlier today (June 22), the U.S. Food... + más
The Boston Globe USA Business June 22, 2023
The Food and Drug Administration on Thursday approved the first gene therapy for Duchenne muscular dystrophy, a $3.2 million one-time treatment from a Cambridge biotech for a deadly inherited neuromuscular disorder that afflicts more than 10,000 boys and young men in the US.But... + más
FDA advisers recommend accelerated approval of a Sarepta gene therapy | The Boston Globe
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
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