News

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 ...
The American Journal of Managed Care1d
FDA Recommends Sarepta Resume DMD Gene Therapy Shipments for Ambulatory Patients
Shipment of delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), will resume following a ...
India Today on MSN1d
What forced Vinay Prasad to exit top job at US FDA in just 3 months
Indian-origin scientist Vinay Prasad resigned as the FDA's vaccine and gene therapy chief in just three months after backlash ...
PharmExec2d
Vinay Prasad’s FDA Departure Follows Controversy Over Drug Rejections
The FDA’s top vaccine and gene therapy official resigned amid heightened scrutiny over recent drug approval decisions and ...
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 ...
Just The News2d
FDA official resigns after pressure from White House, conservative criticism: reports
Dr. Vinay Prasad faced criticisms from disease advocates and conservatives over his decision to pause a muscular dystrophy ...
Chemistry World2d
Sarepta gene therapy deaths highlight tragic rare disease dilemma
Regulators block Duchenne muscular dystrophy treatment after fatal side-effects outweigh questionable efficacy ...
Asianet Newsable on MSN2d
FDA Official Who Halted Sarepta’s Gene Therapy Resigns Amid Backlash Over Access To Duchenne Treatment
Vinay Prasad resigned from the FDA after just two months in the role, following backlash over blocking access to Sarepta’s ...
BioWorld3d
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 ...
Opinion
On Sarepta, a Welcome FDA Reversal
After our editorial, the agency relents to allow a Duchenne treatment.
Becker's Hospital Review3d
Death of 8-year-old not tied to Sarepta gene therapy: FDA
The FDA has reported that the recent death of an 8-year-old boy was unrelated to Sarepta Therapeutics’ gene therapy Elevidys and recommended lifting the voluntary hold on the treatment, which is used ...