Gene activity in SMA may be dysregulated, but the way the disease affects genes seems to vary across types of tissues, per a ...

Early diagnosis of rare diseases like Spinal Muscular Atrophy (SMA) is crucial for managing symptoms and improving quality of ...

A three-month-old boy was seen at the outpatient clinic because of reduced spontaneous movements, which his parents had noticed for a few weeks. His legs lay to the side, he barely moved his hands, ...

Despite disappointing results in the genetic neurodegenerative disorder, the drug shows potential for treating obesity.

The authors emphasize the value of subgroup analyses for tracking patterns in spinal muscular atrophy (SMA), as opposed to ...

While taldefgrobep alfa failed to show improved motor function in spinal muscular atrophy, treated patients saw a marked ...

Novartis has made a dramatic return to the Huntington's disease therapy stage by licensing a candidate from PTC Therapeutics ...

A phase 3 trial of Biohaven’s taldefgrobep alfa in spinal muscular atrophy (SMA) has missed its primary endpoint. The biotech ...

Luis's wheelchair is part of his everyday life with spinal muscular atrophy and he faces challenges with mobility and living ...

In India, approximately 4,000 children are born with Spinal Muscular Atrophy (SMA) every year, making it a critical, albeit ...

As the deadline for a ₹974 crore rare disease fund ends, CureSMA urged the government to act swiftly. At a virtual press meet ...