Feb 3, 2025 · Mouth opening unusually small in youngest SMA patients, study finds. Bulbar muscles, those of the face and throat, are affected in spinal muscular atrophy (SMA), and researchers in Germany found that young children with SMA cannot open their mouths as well as age-matched infants and toddlers without this disease.

3 days ago · Last month, the biotechnology company Scholar Rock submitted an application to the U.S. Food and Drug Administration requesting approval of an experimental add-on therapy to treat spinal muscular atrophy (SMA). The investigational drug apitegromab is intended to target a protein in the bloodstream that inhibits muscle growth with …

Jan 3, 2025 · Read this story here. No. 5 — Combination therapy found to synergistically boost SMN production. When combined with a therapy like Spinraza, low-dose risdiplam — the active ingredient in Evrysdi, an approved disease-modifying treatment for SMA — boosted the production of SMN, the protein that’s deficient in SMA, beyond that of each treatment alone.

3 days ago · SMA is a genetic disorder characterized by progressive muscle weakness and wasting.Over the last decades, three new SMA treatments have been approved in the U.S., and all of them have been proven to slow the progression of the disease. However, many people living with SMA, particularly those born before current treatments were made available, continue to experience substantial difficulties ...

COVID-19 made life with SMA more difficult, parents and doctors say. Limited social interaction and difficulties in accessing needed medications and physical therapy were consequences of the COVID-19 pandemic, parents and grandparents of children with spinal muscular atrophy (SMA) in Sweden reported.

SMA News Today is a digital platform intended to provide the spinal muscular atrophy (SMA) community with the most recent news and information on SMA, as well as first-hand community perspectives from our patient and caregiver columnists.. All articles on SMA News Today are original content produced by in-house writers and editors. Occasionally, we seek the opinion of outside experts for ...

Jan 31, 2025 · “With the strength of our Phase 3 data as the foundation of our submission, we look forward to continuing to work closely with the FDA through the review of our BLA [biologics license application] on behalf of patients and families living with SMA,” Jing Marantz, MD, PhD, chief medical officer of Scholar Rock, said in a company press release. ...

Oct 11, 2024 · Data expected to support regulatory applications come from a pivotal group of patients enrolled in the study’s Part B. This involved 75 symptomatic infants with SMA, ages one week to 7 months, who had never been treated with SMA therapies (treatment-naïve), and who received Spinraza at either the approved lower dose or the high-dose regimen.

Dec 18, 2024 · There are several treatment options available for spinal muscular atrophy (SMA), a rare genetic condition characterized by progressive muscle weakness and wasting. The disease mainly affects motor function, but many patients also may experience breathing, swallowing, and speech difficulties, along with bone defects and other symptoms.

Jan 8, 2025 · The addition of the protein, raising SMN protein levels (expression), in combination with an SMN2-targeting therapy was more effective than either of those approaches alone, the team, which created this full-length protein, noted. “These results provide compelling proof-of-principle evidence of the therapeutic potential of our [ready-to-use] protein as an add-on to the existing therapies ...