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 ...

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 ...

Kaitlin Batley, MD, a pediatric neurologist at Children’s Health and assistant professor at UT Southwestern Medical Center, discusses how physicians position different therapies for patients with ...

Novartis is seeking to repurpose its investigational oral spinal muscular atrophy (SMA) drug branaplam to treat Huntington's disease, the Swiss drugmaker said on Wednesday, as it plans a clinical ...

Sales of Biogen’s BIIB key multiple sclerosis (“MS”) drugs like Tecfidera and Tysabri and spinal muscular atrophy (SMA) ...

As their 8-month-old daughter prepares for gene therapy treatment, a Coon Rapids family is bracing for three months of ...

Kaitlin Batley, MD, a pediatric neurologist at Children’s Health and assistant professor at UT Southwestern Medical Center, discusses the role of personalized medicine in spinal muscular atrophy.

Scholar Rock navigates FDA's Complete Response Letter for Apitegromab, aiming for swift resubmission to address spinal ...

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 ...

In a closely watched case, Roche suffered a setback as a court said it would permit a generic version of its rare disease ...

Emma, a third-grader with big plans for her future, received a groundbreaking treatment at just the right time.

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 ...