Child Thriving After Becoming World's Youngest To Receive Gene Therapy For AADC Deficiency
Clinical Trial Success at Cincinnati Children's Supports Recent US FDA Approval for First Therapy to Directly Replace a Gene ...
In RFK Jr., drugmakers face an antagonist with regulatory power
Anxious and bewildered, some drugmakers are reaching out to their lobbyists about how Robert Kennedy’s pending nomination ...
Daily8h
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 ...
Eli Lilly, J&J Sue US Over Rejected Changes To Drug Discounting Program
Eli Lilly became the second drugmaker this week to sue the U.S. government for rejecting their proposed changes to a program ...
MM&M10h
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 ...
STAT13h
FDA commissioner: We need action and higher-quality research on ultra-processed foods
Researchers, public health and health care groups, advocates, industry, and the public must put tackling diet-related disease ...
Pharmabiz18h
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., ...
FDA's New Rules Require TV Drug Ads to Be Clearer, More Direct
The U.S. Food and Drug Administration ( FDA) now requires TV and radio drug ads to be clearer and more direct. The new ...
BioPharma Dive1d
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 Reference1d
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 ...
BioWorld1d
Healing a health divide (that’s long overdue)
At the BioFuture 2024 conference held in New York in November, Seema Kumar, the CEO of Cure, described women’s health as something that has been directed at the “bikini area.” That “bikini” bias ...