SMA

How Have Respiratory Outcomes Changed With New Spinal Muscular Atrophy Treatments?
How Have Respiratory Outcomes Changed With New Spinal Muscular Atrophy Treatments? 1024 575 Mary Bates, PhD
Illustration of lungs on blue silhouette of upper chest on black background

Three new therapies are changing the prognosis for spinal muscular atrophy, but long-term monitoring of respiratory outcomes is still necessary.

read more
An Expanded, Multicenter Look at Gene Therapy for Spinal Muscular Atrophy
An Expanded, Multicenter Look at Gene Therapy for Spinal Muscular Atrophy 1024 575 Mary Bates, PhD
Colorful illustration of gene therapy in action

New study confirms safety and efficacy in children under two years old. In May 2019, the U.S. Food and Drug Administration (FDA) approved a gene replacement therapy for the inherited, progressive neuromuscular disease 5q-linked spinal muscular atrophy (SMA). Approval included all children with SMA under the age of two years; however, the gene therapy had only been…

read more
Phase 1 Study Shows Promise of Gene Replacement Therapy for Spinal Muscular Atrophy Type 1
Phase 1 Study Shows Promise of Gene Replacement Therapy for Spinal Muscular Atrophy Type 1 1024 575 Abbie Roth

The phase 1 clinical study shows that gene therapy extends survival of patients and supports achievement of milestones previously unseen in the natural course of the disease. A one-time intravenous infusion of the high dose of gene therapy extended the survival of patients with spinal muscular atrophy type 1 (SMA1), according to a study published in…

read more