MyChesCo on MSN1d
Revolutionary Gene Therapy Approved: Kebilidi Offers New Hope for Rare Genetic Disorder
The U.S. Food and Drug Administration (FDA) has announced a groundbreaking approval of Kebilidi (eladocagene exuparvovec-tneq ...
Becker's Hospital Review1h
1st gene therapy delivered directly into brain approved
The FDA granted accelerated approved to the first gene therapy directly administered into the brain on Nov. 14. The therapy, Kebilidi, was approved to treat adults and children with aromatic L-amino ...
biopharma-reporter1d
FDA approves first-ever gene therapy to be directly administered to the brain
PTC Therapeutics has received FDA approval for a gene therapy to treat AADC deficiency, a rare genetic disorder that impairs ...
InnovationRx: Trump Picks RFK, Jr. And Dr. Oz For Key Healthcare Roles
InnovationRx is your weekly digest of healthcare news. To get it in your inbox, subscribe here.
WebMD1d
First Brain-Injected Gene Therapy Approved by FDA
The FDA has granted fast-track approval for a groundbreaking gene therapy indicated for a rare genetic disorder called ...
BioPharma Dive5d
PTC wins US approval of gene therapy for fatal enzyme disorder
Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review ...
Monthly Prescribing Reference5d
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 Approves PTC's Kebilidi As First Gene Therapy To Treat AADC Deficiency
The U.S. Food and Drug Administration approved PTC Therapeutics, Inc.'s Kebilidi (eladocagene exuparvovec-tneq) gene therapy for the ...
GlobalData on MSN6d
FDA grants accelerated approval for PTC’s AADC deficiency gene therapy
This therapy is designed to address the needs of children and adults with aromatic L-amino acid decarboxylase (AADC) ...
Daily5d
FDA Approves First Gene Therapy for Treatment of AADC deficiency
The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus ...
FDA Grants Conditional Approval For PTC Therapeutics' Gene Therapy As First Ever With Direct Administration In Brain
On Thursday, the FDA granted accelerated approval to PTC Therapeutics, Inc.’s (NASDAQ:PTCT) gene therapy for AADC deficiency, ...
Nasdaq5d
FDA Approves PTC's Kebilidi As First Gene Therapy To Treat AADC Deficiency
(RTTNews) - The U.S. Food and Drug Administration approved PTC Therapeutics, Inc.'s Kebilidi (eladocagene exuparvovec-tneq) gene therapy for the treatment of aromatic L-amino acid decarboxylase or ...