Hovanesian: I would have expected a more rapid uptake of these drugs. The problem was that the first drug approved, Syfovre, had some issues with very rare, but often blinding occlusive vasculitis.

MRI imaging scans can track muscle changes in SMA children given Spinraza, a study found, with better results seen with early ...

Panelists discuss how supportive care for patients with spinal muscular atrophy (SMA) includes rehabilitation, respiratory ...

Spinraza (nusinersen) treatment helped school-age children with SMA be more independent in tasks involving their arms and ...

Subjects receiving the therapy demonstrated clinically meaningful enhancements in physical and muscle function.

Rainbow Children's Hospital in Secunderabad has successfully administered gene therapy to its eighth patient with Spinal ...

Cancer cachexia is a devastating condition that affects up to 80% of advanced cancer patients and causes approximately 2 million deaths worldwide annually. Cancer cachexia is characterized by ...

22. Apitegromab is an investigational treatment aimed at improving motor function for people living with spinal muscular atrophy. Scholar Rock said it has also submitted and received validation ...

In a hopeful step for medicine, a 2-and-a-half-year-old child born with spinal muscular atrophy (SMA) has shown no symptoms ... case of prenatal therapy that holds promise for future treatment of the ...

Completed Phase 2 enrollment with randomization of 51 subjects into treatment and control arms Phase 1/2 study ... modifier gene therapy candidate being developed for geographic atrophy (GA), an ...

There is no cure for spinal muscular atrophy, but treatments can stop its progression and often lead to some improvement. However, Manitoba has refused to pay for the hugely expensive treatment ...