The recent availability of 3 FDA-approved treatments for patients with spinal muscular atrophy (SMA) has dramatically altered patient outcomes and provided a wealth of new avenues for SMA research. A ...
Gene therapy is linked to better nonmotor outcomes in spinal muscular atrophy type 1 compared to first-line nusinersen, new ...
Access to timely treatment for spinal muscular atrophy (SMA) remains a challenge, with barriers including delays in diagnosis, frequent treatment needs, and insurance coverage gaps, which impact ...
After up to 4.8 years of continuous treatment with SPINRAZA, 100 percent of children treated pre-symptomatically were alive, and none require permanent ventilation Patients continued to maintain and ...
The first program I want to go over is the use of taldefgrobep alfa, which is being developed in the phase 3 RESILIENT study for the treatment of patients with spinal muscular atrophy. Before going ...
Imagine a child born in a family with a rare disease, whose treatment would cost more than 17 crore for a single dose. The ...
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today new 5-year data confirming the sustained efficacy and safety profile ...
Medical researchers have uncovered a novel mechanism that leads to motor neuron degeneration in spinal muscular atrophy (SMA). This discovery offers a new target for treatment that overcomes important ...
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, ...
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 ...