Perspectives of patients with spinal muscular atrophy (SMA) and their caregivers should be weighed before making decisions around SMA-enhancing treatments.

SMA type is a strong predictor of some oral functioning in affected patients | image credit: RTimages - stock.adobe.com

Oral Function Impairment Varies Across Adult Patients With SMA

By Karen Jacobson-Sive

October 28th 2024

Oral function should be measured routinely to determine individual bulbar involvement stages in spinal muscular atrophy (SMA).

SMA spinal muscular atrophy | Image Credit: © Rochu_2008 - stock.adobe.com

Protein Level Correlates With Pediatric SMA Severity, Treatment Response

By Jared Kaltwasser

October 17th 2024

Investigators say thrombospondin-4 levels were reduced in patients with symptoms of spinal muscular atrophy (SMA), but levels of the protein increased after therapy.

SMA puzzle pieces | image credit: Dzmitry - stock.adobe.com

Muscle-Directed Therapy Apitegromab Meets Primary End Point in Phase 3 SAPPHIRE Trial of SMA

By Marco Meglio

October 17th 2024

Patients aged 2-21 receiving apitegromab showed clinically meaningful motor function improvements, with a favorable safety profile consistent with long-term data, as Scholar Rock prepares for US and EU regulatory submissions in Q1 2025.

...

3 4 5 6 7

2 Commerce Drive
Suite 100
Cranbury, NJ 08512

Do Not Sell My Information

Terms & Conditions