AskBio Receives FDA Rare Pediatric Disease and Orphan-Drug Designations for AB-1003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9
First patient dosed in Phase 1/Phase 2 LION-CS101 trial of AB-1003 in August 2023, with enrollment continuing Asklepios ...
onmanorama4d
Actor Nepoleon's son's wedding: What is muscular dystrophy?
According to the Mayo Clinic, muscular dystrophy is a group of diseases that cause loss of muscle mass and progressive ...
CUMBERLAND PHARMACEUTICALS RECEIVES FDA ORPHAN DRUG AND RARE PEDIATRIC DISEASE DESIGNATIONS FOR NEW TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX), a specialty pharmaceutical company, announced today that the United States (U.S.) Food and Drug Administration (FDA) granted Orphan Drug Designation and ...
Wave Life Sciences’ Q3 2024 Advances in RNA Therapies
Wave Life Sciences Pte. Ltd ( (WVE) ) has released its Q3 earnings. Here is a breakdown of the information Wave Life Sciences Pte. Ltd ...
Dyne Therapeutics' Q3 Preview: High-Risk, High-Reward Path In Muscle Diseases
Investors consider Dyne Therapeutics' treatments for muscle diseases and strong cash position as a speculative Buy ...
Wave Life Sciences price target raised to $36 from $17 at Truist
Truist analyst Joon Lee raised the firm’s price target on Wave Life Sciences (WVE) to $36 from $17 and keeps a Buy rating on the shares.
Pharmabiz5d
US FDA grants orphan drug & rare paediatric disease designations to Cumberland Pharma’s Ifetroban to treat cardiomyopathy associated with DMD
Nashville, Tennessee Friday, November 8, 2024, 13:00 Hrs [IST] ...
Sarepta Therapeutics: BLA Filing Of SRP-9003 For LGMD Is The Next Step
Sarepta Therapeutics is advancing SRP-9003 gene therapy for LGMD, with phase 3 study results expected in early 2025. Learn ...
The Pharma Letter6d
FDA Rare Pediatric Disease status for AskBio’s AB-1003
German pharma major Bayer’s independently operated subsidiary Asklepios BioPharmaceutical (AskBio) today announced that AB-1003 (also known as LION-101) has received rare pediatric disease designation ...
Medscape6d
Therapy Insight: Cardiovascular Complications Associated With Muscular Dystrophy
Although often considered to be diseases of childhood, adult forms of muscular dystrophy occur and represent a spectrum of disease. Muscular dystrophies are a genetically heterogeneous group of ...
Stevens Institute of Technology45m
NIH Awards $2 Million to Stevens, Columbia to Monitor Neuromuscular Disorders
In-shoe sensors, AI and biomarkers will monitor muscular dystrophy and atrophy in children, adolescents and adults — with ...