News

FDA’s Reversal on Gene Therapy Helps My Son
Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.
PTC Therapeutics: Uncertainty Abounds Ahead Of Q2 Earnings - I'm Upgrading To 'Hold'
PTC Therapeutics faces pivotal FDA decisions for approvals that could potentially transform their long-term revenues. Click ...
Amazon S3 on MSN2d
The story of Sarepta's Duchenne gene therapy
The last couple of weeks has been particularly distressing for Duchenne muscular dystrophy patients and their families. The ...
Healio2d
Oncologist who served as FDA’s chief medical officer, top vaccine official departs agency
A controversial hematologist-oncologist who joined FDA this spring and quickly became its top regulator has left the ...
STAT2d
STAT breaks down the confusing, heartbreaking Sarepta saga
In the latest episode of STATus Report, Alex Hogan breaks down the confusing, heartbreaking saga of Sarepta and its gene ...
InnovationRx: Trump’s EU Tariff Deal Will Add $13 Billion To Drug Costs
In this week’s edition of InnovationRx, we look at Halle Berry’s menopause startup, Ambience Healthcare’s $243 million ...
The New York Sun2d
FDA’s Top Regulator Is Out Amid MAHA Backlash Over Blocking Access to a $3.2 Million Per Dose Therapy for Rare Disorder in Boys
Vinay Prasad, who came under fire from patient advocacy groups and Laura Loomer, is gone after less than three months on the ...
BioSpace2d
Sarepta Gets Reprieve, RFK Jr.’s New Changes, Roche’s Alzheimer’s Comeback and Q2 Earnings
Sarepta’s Elevidys is back on the market for ambulatory patients with Duchenne muscular dystrophy, Health Secretary Robert F. Kennedy Jr. reportedly plans to dissolve the U.S. Preventive Services Task ...
Top FDA Official Appointed By Trump Admin Departs After Three Months
A spokesperson for the Health Department has said that Vinay Prasad did not want to be a "distraction to the great work of the FDA in the Trump administration." ...
Just The News2d
FDA allows distribution of muscular dystrophy drug again after public criticism
The agency’s now-reversed decision to halt distribution of a gene therapy for children suffering from muscular dystrophy ...
Controversial FDA official Dr. Vinay Prasad departs agency
Dr. Vinay Prasad, the controversial critic of the US Food and Drug Administration who took a top role at the regulatory ...
BioWorld2d
Apellis’ Empaveli approved in US for two rare kidney diseases
The approval to reduce proteinuria for those 12 and older with either of the two severe and rare kidney diseases came on the July 28 PDUFA date, a little more than four years after the company won ...