Treatment with a high-dose regimen of Spinraza safely improved motor function in children with SMA, according to data from the DEVOTE trial.

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 ...

Shared decision-making between patients and healthcare providers is essential for optimal SMA treatment outcomes and quality of life. Early treatment initiation is crucial for infants, while older ...

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 ...

Findings from DEVOTE support clinical benefits of the high-dose regimen of nusinersen (50 mg and 28 mg) in both ...

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 ...

Jesy Nelson is keeping a positive approach after her 8-month-old Twin baby girls were diagnosed with severe muscular ...

In a hopeful step for medicine, a 2-and-a-half-year-old child born with spinal muscular atrophy (SMA) has shown no symptoms ...

The first prenatal treatment for spinal muscular atrophy showed promise in a single case report. Risdiplam was given to the mother during pregnancy and to the child after birth. More than 2 years ...

It is classified as one of the rare diseases, yet it affects approximately one in 7,744 live births in India. There is no cure for SMA; it can only be managed, and the progression can either be ...

Little Mix alum Jesy Nelson is opening up about her 9-month-old twin daughters’ battle with a severe muscle disease. Nelson, 34, expressed hope that her baby girls, Ocean Jade and Story Monroe, would ...