Spinal Muscular Atrophy

Updates in Treatment of Adult Spinal Muscular Atrophy

Challenges and Considerations in Treatment of Adult Spinal Muscular Atrophy

Key opinion leader discusses considerations for adult patients with spinal muscular atrophy (SMA) including disease burden, treatment evaluation, and the use of real-world evidence.

Jacqueline Glascock of Cure SMA Discusses FDA-Approved SMA Treatments & Dissects Combination Therapy

November 17, 2021

Managed Healthcare Executive® recently interviewed Jacqueline Glascock, Ph.D., the director of research programs at Cure SMA, an nonprofit organization in Elk Grove, Illinois, that funds research into spinal muscular atrophy (SMA) and advocates for families and patients. In this video, Glascock discusses SMA and her organization, the three current therapies for the disease, and a combination therapy approach to the disease.

Metabolic Urine Analysis May Help Diagnose, Predict SMA

November 16, 2021

A new study suggests clinicians might be able to use metabolic “fingerprints” to better understand the likely severity of individual cases.

DNA from Saliva has Potential for Detecting SMA

November 15, 2021

Investigators in Japan have developed a new, noninvasive method for screening for spinal muscular atrophy using saliva analyzed with conventional PCR as an alternative to blood-based genetic screening.

European Study Confirms Safety and Efficacy of Zolgensma in SMA

October 14, 2021

This European study enrolled patients with more severe cases of spinal muscular atrophy than a similar study conducted in the U.S.

Study Looks At Novel Administration of Spinraza

October 14, 2021

A new method for delivering this treatment drug for spinal muscular atrophy offers an option for patients with advanced cases of the disease. But investigators found that the new method may also carry risks of mechanical failure and infection.

What Is in the SMA Pipeline?

September 28, 2021

Small biotech companies such as Scholar Rock and Cytokinetics have treatments in late-stage trials for spinal muscular atrophy. Novartis and Biogen also have products in the pipeline.

An Overview of the SMA Treatment Landscape

September 27, 2021

The FDA has approved three targeted therapies for spinal muscular atrophy. They have hefty price tags so cost and affordability are live topics.

Phase 2 Trial Drug Apitegromab Shows Progress for Spinal Muscular Atrophy Patients

March 17, 2021

The phase 2 TOPAZ trial demonstrated treatment is safe and improves motor function in patients with SMA.

Spinal Muscular Atrophy Drug Risdiplam Proves Success in Infants

March 02, 2021

Patients treated with risdiplam at 12 months demonstrated significant improvement in survival and developmental milestones, with 19 of 21 (90%) infants able to survive without permanent ventilation and 7 (41%) infants able to sit without support for at least 5 seconds.

FDA Approves Evrysdi, Third Drug for Spinal Muscular Atrophy

August 10, 2020