Yahoo Finance7h
REGENXBIO to Host Webcast Discussing Pivotal Program and First Functional Data from the AFFINITY DUCHENNE® Trial of RGX-202
These updates will also be presented at the American Society of Gene and Cell Therapy and Muscular Dystrophy Association 2024 Breakthroughs ... including Novartis' Zolgensma ® for children with spinal ...
onmanorama5d
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 ...
charitytoday.co.uk6h
Charity reveals cost of living impact on neuromuscular conditions
Over half of the people who contributed to the report (53%) said they feel financially insecure. 17% of those reported ...
Medscape10d
New Drug Options Abound for Duchenne Muscular Dystrophy
When Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, pediatric neurologist Nancy L. Kuntz, MD, was a fellow ...
The Korea Bizwire7d
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 ...
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 ...
Muscular Dystrophy News5h
FDA OKs trial to test gene therapy candidate in LGMD children
The FDA has given the green light to a clinical trial testing ATA-200, a gene therapy for limb-girdle muscular dystrophy ...
Medscape7d
Association of Duchenne Muscular Dystrophy With Autism Spectrum Disorder
We hypothesize that Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder co-occur with a greater than random frequency. In this study, we set out to reject the ...
Santhera Enters into Exclusive Supply Agreement for AGAMREE® (Vamorolone) with Ali Al Suwaidi Trading Establishment (ASTE) in Qatar
Pratteln, Switzerland, November 12, 2024 – Santhera Pharmaceuticals (SIX: SANN) announces the signing of an exclusive supply agreement with Ali Al Suwaidi Trading Est. (ASTE) for the treatment of ...