December 16th 2021
Early treatment helps improve the efficacy of treatment of spinal muscular atrophy, according to a comprehensive review article published earlier this month.
December 9th 2021
In this diagnostic proof-of-concept trial, an emerging imaging approach that uses a handheld laser demonstrated the ability to visualize muscle degeneration in children with SMA.
November 16th 2021
A new study suggests clinicians might be able to use metabolic “fingerprints” to better understand the likely severity of individual cases.
November 15th 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.
October 14th 2021
This European study enrolled patients with more severe cases of spinal muscular atrophy than a similar study conducted in the U.S.
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.
Study Looks At Novel Administration of Spinraza
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?
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
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
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
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.
Nusinersen in Adults With 5Q SMA
FDA Approves Evrysdi, Third Drug for Spinal Muscular Atrophy
FDA Updates: Assays Used in Cancer Care Get Green Light
Adaptive Biotechnologies’ clonoSEQ gets third approval; FDA also acts on first at-home treatment for spinal muscular atrophy.
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