The Pharma Letter22h
FDA nod for AADC deficiency gene therapy from PTC
The US Food and Drug Administration (FDA) has granted accelerated approval of PTC Therapeutics’ gene therapy for the ...
Pharmabiz9h
PTC Therapeutics’ gene therapy, Kebilidi to treat AADC deficiency receives US FDA approval
PTC Therapeutics’ gene therapy, Kebilidi to treat AADC deficiency receives US FDA approval: Warren, New Jersey Friday, November 15, 2024, 10:00 Hrs [IST] PTC Therapeutics, Inc., ...
emjreviews.com21h
FDA approves first gene therapy delivered directly to the brain
The drug is a treatment for a rare and fatal genetic disorder called aromatic l-amino acid decarboxylase (AADC) deficiency, ...
Monthly Prescribing Reference16h
Kebilidi, a Gene Therapy for AADC Deficiency, Gets Accelerated Approval
The Food and Drug Administration (FDA) has granted accelerated approval to Kebilidi™ (eladocagene exuparvovec-tneq) for the treatment of adult and pediatric patients with aromatic L-amino acid ...
FDA Grants Conditional Approval For PTC Therapeutics' Gene Therapy As First Ever With Direct Administration In Brain
PTC Therapeutics' Kebilidi gains FDA accelerated approval as the first brain-administered gene therapy in the U.S. for AADC ...
GlobalData on MSN22h
FDA grants accelerated approval for PTC’s AADC deficiency gene therapy
The US Food and Drug Administration (FDA) has granted accelerated approval for PTC Therapeutics’ KEBILIDI, the first gene ...
FiercePharma22h
PTC wins FDA approval for first brain-delivered gene therapy Kebilidi
The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...
PTC Therapeutics price target raised to $45 from $43 at Barclays
Barclays raised the firm’s price target on PTC Therapeutics (PTCT) to $45 from $43 and keeps an Equal Weight rating on the shares. After ...
Le Lézard23h
GiangisKitchen.com Shares Mouth-Watering Recipes that Can be Easily Prepared Days Before Thanksgiving
Popular recipe site GiangisKitchen.com has published a unique selection of recipes for delicious Thanksgiving appetizers, ...
MM&M29m
Rx Rundown: Roche, Metsera, Intercept Pharmaceuticals and more
AstraZeneca and Amgen announced that a Phase 3 trial of Tezspire (tezepelumab) showed a statistically significant and ...
Le Lézard23h
Leadership Expert Ann Bowers-Evangelista: The Four Pillars of Leadership Endurance
Many businesspeople wonder how they will have enough energy to lead for the next 20 to 30 years because life feels like a marathon. "In my coaching and consulting practice, I often encounter leaders ...