Patients treated with OAV101 IT showed an increase from baseline in HFMSE total score compared with sham control. Topline data were announced from a phase 3 trial evaluating intrathecal onasemnogene ...
Spinal Muscular Atrophy (SMA) causes progressive muscle weakness and, without treatment, can limit life expectancy to just ...
"Decreasing AMMO [active maximal mouth opening] over time is seen most often in patients with SMA type 2 with an estimated reduction of almost 1 mm per year...At this point, we propose that ...
Study Validates the Positive Effects of Nusinersen on Bone Health in Patients With SMA Types 2 and 3
In a multidisciplinary follow-up study, researchers analyzed bone health decline and the effects of nusinersen in patients with spinal muscular atrophy (SMA) types 2 and 3. SMA is a progressive, ...
Some patients with later-onset spinal muscular atrophy (SMA) type 2 and type 3 had improved motor function when the investigational monoclonal antibody apitegromab was added to their treatment, the ...
PHILADELPHIA--(BUSINESS WIRE)--Exegenesis Bio, a rapidly growing global genetic medicines company, is pleased to announce the presentation of clinical efficacy and safety data from its EXG001-307 ...
Biomarkers for SMA were identified using LC-MS and machine learning, revealing differences in protein expression across SMA types. CNTN1 and NRXN3 proteins showed potential as prognostic biomarkers, ...
Scotland launches a newborn screening pilot for spinal muscular atrophy (SMA) via the heel prick test in Glasgow, aiming for ...
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