Spinal muscular atrophy (SMA) is a group of serious, progressive diseases that destroys motor neuron cells. SMA treatment aims to reduce symptoms and slow or stop the progression of the disease. SMA ...

A gene therapy candidate extended survival in a mouse model of SMARD1, supporting its testing in a clinical trial for this ...

At her eight-month pregnancy check-up, Lizmara Figueroa met with a new doctor. As he walked out the door, he made a parting comment. “He says, ‘Oh, just so you know, you’re a carrier for (spinal ...

At her eight-month pregnancy check-up, Lizmara Figueroa met with a new doctor. As he walked out the door, he made a parting comment. “He says, ‘Oh, just so you know, you’re a carrier for (spinal ...

Spinal muscular atrophy (SMA) is a severe neurological disease for which there is presently no cure, although current therapies can alleviate symptoms. In the search for better treatment options, ...

The tiny zebrafish is helping researchers rapidly determine whether a newborn's genetic mutation is likely to cause spinal ...

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today presented positive results from the primary analysis of the ongoing RAINBOWFISH ...

MISSISSAUGA, ON, April 15, 2021 /CNW/ - Hoffmann-La Roche Limited (Roche Canada) today announced that Health Canada has granted EVRYSDI Ⓡ (risdiplam) market authorization for the treatment of spinal ...

DUBLIN--(BUSINESS WIRE)--The "Spinal Muscular Atrophy (SMA) Treatment Market Size, Share & Trends Analysis Report By Route of Administration (Oral, Intrathecal), By Treatment (Gene Therapy, Drug), By ...

Regina Trollmann, MD, of the Division of Pediatric Neurology, Department of Pediatrics, Friedrich-Alexander-University of Erlangen-Nürnberg, Erlangen, Germany, and colleagues, did a retrospective ...