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 ...
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 ...
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 ...
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 ...
'Enjoy the world while you can,' says teen with MD
Michael McGrath, from the charity, says the days out, called "muscle warrior days" offer "highly personalised" support for ...
Medscape6d
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 ...
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] ...
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 ...
CTV News1d
Windsor Paralympic champion crowned inspirational speaker of the year
Windsor Paralympic champion, Danielle Campo, has been crowned the number one Inspirational Speaker of the Year for 2024 at ...
Independent.ie5d
Wexford woman known for her kindness and strength is named carer of the year
A Clonard woman who has dedicated her life to providing full-time care to her son, as well as being a source of help and ...
This 'beloved holiday tradition' is returning to Indianapolis
INDIANAPOLIS — The Christmas Night of Lights, the largest drive-thru light show in Indianapolis, is returning this holiday ...