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

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

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

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

Roche to present latest scientific advancements from its neuromuscular portfolio at Muscular Dystrophy Association 2025 conference: Basel Tuesday, March 18, 2025, 11:00 Hrs [IST] ...

And then, of course, we have disease-modulating therapies that need to be initiated as soon as possible when somebody has an established diagnosis of SMA to prevent irreversible motor neuron loss.

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

With ongoing breakthroughs in gene and RNA therapies, coupled with regulatory encouragement and AI-based innovation, the market for treating rare neurological diseases is on the cusp of a ...

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 ...

Spinal muscular atrophy type 1 is a genetic disorder that typically appears within the first six months of life, causing ...

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

Dr Anup Rawool, Consultant in Clinical and Cancer Genetics at Sahyadri Hospitals, Pune, discusses innovative therapies and ...