Yahoo Finance1d
IND for ATA-200, a Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2C/R5 (LGMD2C/R5), cleared to proceed by FDA
ATA-200 is a one-time gene therapy for the treatment of γ-sarcoglycan related limb-girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5), a serious and debilitating condition that affects children and ...
Traumatized by war, hundreds of Lebanon's children struggle with wounds both physical and emotional
Ghassan Abu Sittah, a renowned British-Palestinian surgeon who is also treating Hussein, sees that long road ahead. This is ...
radaronline4d
Jerry Lewis' Bitter Battle With Muscular Dystrophy Charity Revealed: Why Do-Gooder Bosses Dumped 'Egomaniac'
Jerry Lewis' childhood friend and former manager revealed the real reason he got the boot from the Muscular Dystrophy telethon after 45 years of hosting.
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 ...
The Lancet7d
A contemporary analysis of the Australian clinical and genetic landscape of spinal muscular atrophy: a registry based study
Despite diagnostic and therapeutic advances, mortality and the multi-systemic health impact of SMA continue to be experienced ...
Medscape9d
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 ...
WISH-TV10d
Indiana licensing program ‘putting a dent’ in special educators shortage
Discover how the I-SEAL program is helping teachers in Indiana obtain special education licenses for free, addressing a critical shortage in the field.
Nationwide Children's tops tech commercialization in Ohio for fifth year
For the fifth year in a row, Nationwide Children's Hospital made the most income statewide from licensing its technology, according to an annual nationwide survey.
Muscular Dystrophy Association Shines a Spotlight on National Family Caregivers Awareness Month in November
Brooke has been unstoppable raising awareness and funds for ALS research while introducing our entire family to a community of support with the Muscular Dystrophy Association.” Challenges Rare Disease ...
SciTech Daily15d
New Model Unveils Liver Secrets of Myotonic Dystrophy Type 1
In muscular dystrophy type 1, mutated DNA begets toxic RNA that alter liver function, including susceptibility to fatty liver disease and hypersensitivity to drugs. Understanding these effects is ...
BioWorld16d
MDA’s Kickstart program announces orphan drug designation for congenital myasthenic syndrome
The Muscular Dystrophy Association (MDA)’s Kickstart program has announced receipt of both U.S. orphan drug and rare pediatric disease designations in support of work for congenital myasthenic ...
The New Indian Express17d
ARDO, a beacon of hope for muscular dystrophy patients
Speaking to TNIE, Sobharani Sunkara, a patient herself as well as the ARDO Founder and Executive Director, said, “We want to show children with muscular dystrophy that life is still full of ...