Inozyme Pharma to Present Recently Announced Interim Data for INZ-701 in Infants and Young ...
Title: Impact of the Enzyme Replacement Therapy, INZ-701, in Children with ENPP1 Deficiency: Experience from an Expanded Access Program Presentation Number: 10 – Poster Session Date: Saturday, ...
The Lancet14 小时
Vitamin D and acute respiratory infections: a definitive answer?
Acute respiratory infections (ARIs) are a common cause of illness in children younger than 16 years and adults older than 65 ...
Medindia9 小时
FDA Approves First Treatment for Cerebrotendinous Xanthomatosis, a Rare Lipid Storage Disease
"The FDA is dedicated to supporting new drug development for rare diseases including very rare metabolic diseases like cerebrotendinous xanthomatosis," said Janet Maynard, M.D., M ...
FiercePharma18 小时
After asset purchase from Travere, Mirum's Ctexli crosses FDA finish line in rare metabolic ...
The approval makes chenodiol—already cleared for decades to treat radiolucent gallstones under the brand name Chenodal—the ...
1MIRM : FDA Approves Mirum Pharmaceuticals' Drug As First For Genetic Disorder...
Mirum Pharmaceuticals' Ctexli wins FDA approval for CTX, a rare lipid storage disease. The company projects 2025 global sales of $420–$435 million.
Cell And Gene Therapies In Rare Disorders Market To Skyrocket Across The 7MM During The ...
The cell and gene therapies in rare disorders market is expected to grow at a significantly high rate during the forecast period (2025–2034) and main ...