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Antibodies to Spinal Muscular Atrophy Gene Therapy Vector May Dissipate, Allowing More Children to Receive Treatment
 Antibodies to Spinal Muscular Atrophy Gene Therapy Vector May Dissipate, Allowing More Children to Receive Treatment 1024 681 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES
sleeping infant

A review of antibody titers for SMA patients revealed that levels may decrease with time, potentially enabling delayed dosing for children who otherwise might have been excluded from life-saving gene therapy. Adeno-associated viral (AAV) vectors are used to deliver gene therapies such as onasemnogene abeparvovec-xioi (ZOLGENSMA®), a recently approved gene therapy for spinal muscular atrophy…

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
By: 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.

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
By: 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…

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 Miller
By: Abbie Miller

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…