GlobalData on MSN8h
Perspectum awarded $250k FDA grant for rare disease research
The grant will support Perspectum’s research into the development of a novel biomarker for primary sclerosing cholangitis ...
Hosted on MSN2d
How logistics shape the success of rare disease trials
Both events are taking place simultaneously 19–20 November in Brussels, Belgium. As interest in rare disease drug development ...
FDA grants orphan drug designation to potential treatment of STXBP1 developmental and epileptic encephalopathy
Research that was carried out by Baylor College of Medicine and Texas Children's Hospital doctors has led to the U.S. Food ...
JD Supra10d
New Momentum for a Time-Limited Conditional Approval Pathway for Rare Disease Drugs
On October 4, 2024, a US House version of the revised Promising Pathway Act (PPA) 2.0 was introduced, sponsored by Rep. Bruce Westerman (R-AR).
JD Supra8d
FDA Amplifies Cross-Center Collaboration with the Creation of the Rare Disease Innovation Hub
Roughly one out of every ten people in the United States are affected by rare diseases. Many of these conditions are life‑threatening, and most ...
AskBio Receives FDA Rare Pediatric Disease and Orphan-Drug Designations for AB-1003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9
(AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that AB-1003 (also known as LION-101) has received rare pediatric disease ...
GEN1d
AI Collaboration Between Sanofi and Healx Will Target Novel Rare Diseases Indications
Healx will use its AI-based drug discovery tech to analyze proprietary Sanofi compound data and identify potential rare disease targets.
fingerlakes110d
The Role of AI in Drug Development Revolution: What It Means for Healthcare?
The influence of AI in drug development has gained such importance that it has been termed a revolution. Let’s explore how ...
Actuate announces FDA rare pediatric disease designation to elraglusib
Actuate Therapeutics (ACTU) announced that the U.S. Food and Drug Administration, FDA, has granted rare pediatric disease designation to ...
Yahoo Finance8d
CUMBERLAND PHARMACEUTICALS RECEIVES FDA ORPHAN DRUG AND RARE PEDIATRIC DISEASE DESIGNATIONS FOR NEW TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
"For Duchenne muscular dystrophy, a devastating genetic disorder affecting young boys, securing both Orphan Drug and Rare Pediatric Disease Designations for Ifetroban from the FDA is a critical ...