Spinal Muscular Atrophy

November 5th 2024

In a response to a survey, caregivers of people with spinal muscular atrophy identified the risk of severe adverse events and the need for permanent ventilation as the most important factors in treatment decisions. Access to treatment, including cost and availability, ranked third.

October 25th 2024

Some prior research had shown that gene therapy would not lower the amount of time children spend on ventilator support.

October 15th 2024

Two years after treatment, both girls show normal motor function and no signs of spinal muscular atrophy symptoms.

October 9th 2024

Apitegromab works by targeting a protein called myostatin that limits muscle growth and is designed to be used alongside existing spinal muscular atrophy (SMA) treatments.