Por: RTTNews 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 with Duchenne muscular dystrophy.SRP-9001 is an investigational gene therapy for Duchenne being developed in partnership with Roche.The BLA is submitted for accelerated... + full article
ABC News USA Business October 01, 2022
WASHINGTON -- When patients with a deadly diagnosis and few treatment options have tried to get unapproved, experimental drugs, they have long faced a dilemma: Who will pay?Responsibility for funding so-called compassionate use has always fallen to drugmakers, though many are... + más
NIH to fund unproven ALS drugs under patient-backed law | WPLG Local 10
NIH to fund unproven ALS drugs under patient-backed law | Associated Press
WPLG Local 10 USA Politics October 01, 2022
WASHINGTON – When patients with a deadly diagnosis and few treatment options have tried to get unapproved, experimental drugs, they have long faced a dilemma: Who will pay?Responsibility for funding so-called compassionate use has always fallen to drugmakers, though many are... + más
NIH to fund unproven ALS drugs under patient-backed law | ABC News
The Boston Globe USA Business September 30, 2022
The Food and Drug Administration on Thursday approved a controversial new drug for ALS from a Cambridge company, even though agency scientists had questioned whether it would help patients with the devastating disease and a panel of independent medical experts initially... + más
Highly debated Amylyx Pharmaceuticals ALS drug wins FDA approval | New York Post
ALS drug wins FDA approval despite questionable data | Portland Press Herald
Forbes USA Life September 30, 2022
Diageo Elusive ExpressionsDiageo There are a few big days every year for whisky fans, and one of them for me is always the announcement of Diageo’s Special Releases for the year. The yearly collection spans Diageo’s portfolio of single malts and typically offers some pretty... + más
The Bicester Collection, Supporting Sustainability And Emerging Fashion Designers During Milan And Paris Fashion Weeks | Forbes
Daniel Rosenberry Turns To The Extraordinary For Schiaparelli’s SS23 Collection | Forbes
ABC7 USA Health September 25, 2022
After discovering how little information was available on Duchenne, these parents started a nonprofit, Cure Duchenne, now funding research toward a cure and resources for other families going through the same journey.We funded over 44 research projects. Sixteen of those have... + más
Sarepta Submits BLA For SRP-9001 To Treat Ambulant Patients With Duchenne Muscular Dystrophy | RTTNews
Starting a Revolution Isn’t Enough | The Atlantic
The Atlantic USA Science September 24, 2022
CRISPR is changing the world—but it can do more.Erik Carter / The AtlanticSeptember 12, 2022Two years ago, I was working on my laptop in an airport lounge in Newark, New Jersey, when I glanced up and saw a couple walking with their two boys. The younger boy slowly made his way... + más
Teenagers on Revolution roster have provided a spark that bodes well for the future | The Boston Globe
‘I Think the Women Are Winning’ | The Atlantic
The Advocate USA Health September 17, 2022
The concierge medical market in the U.S. is expected to reach $13.3 billion by 2030, up from $6.1 billion in 2022, according to Research and Markets reports.What is concierge medicine? It’s a subscription-based model of primary care medicine. Most patients pay a membership fee... + más
Poll: Americans give health care system failing mark | 10 WBNS
May is Mental Health Awareness Month: COVID may have normalized mental health struggles | The Advocate
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