Scholar Rock’s spinal muscular atrophy (SMA) therapy has shown benefit in muscle function in children under 12 in a pivotal ...

A multidisciplinary approach, early diagnosis, and comprehensive caregiver education are crucial in effectively managing ...

Scholar Rock will present new Phase 3 SAPPHIRE trial data on apitegromab at the 2025 MDA conference, highlighting efficacy ...

New Evrysdi five-year data from the SUNFISH study showed continued stabilisation of motor function in a broad population of ...

The challenge of the obesity epidemic is widespread and one that even pediatricians face constantly,” a Connecticut doctor ...

The author highlights the real-life impacts on the residents of Knoxville and East Tennessee from Trump administration cuts to the NIH.

The parents were both known carriers of SMN1 gene mutations that raised the risk of SMA, and sadly had previously had a child born with the disease who died at 16 months of age. Genetic testing of ...

Leading medical professionals dealing with patients suffering from Spinal Muscular Atrophy (SMA) have urged the c ...

More than 2 years later, the child shows no features of the disease. The first prenatal therapy for spinal muscular atrophy (SMA) showed promising results, a case report indicated. More than 2 ...

Exercise can make a significant difference for patients with SMA by working alongside other medical advancements to improve their quality of life.

SMA patients want PROMs that focus on more than physical function, and that also assess mental health, sexual function, sleep ...