Kerala's Health Department extends free Risdiplam supply to Spinal Muscular Atrophy patients up to 25 years, broadening the previous limit of 12 years. This initiative under the CARE program, launched ...

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 ...

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 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 ...

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 ...

The lab of Yongchao C. Ma, PhD, at Stanley Manne Children’s Research Institute at Ann & Robert H. Lurie Children’s Hospital of Chicago uncovered a novel mechanism that leads to motor neuron ...

Panelists discuss how combination therapies targeting different aspects of spinal muscular atrophy (SMA), such as myostatin inhibitors (targeting muscle) alongside SMN protein-enhancing treatments, ...

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, ...

In a major policy decision announced on Rare Disease Day, Kerala''s Health Department has extended free access to the high-cost drug Risdiplam for patients diagnosed with Spinal Muscular Atrophy (SMA) ...

Responding to the appeal, Lokesh said SMA Type-1 is a debilitating condition that gradually weakens muscles and affects vital functions. He said it was now his responsibility to help raise the ...