In ‘Care Work,’ Grief Is the Garden of Our Dreams
Many, many weeks ago, I said I was going to read “Care Work: Dreaming Disability Justice” by Leah Lakshmi Piepzna-Samarasinha. In a month. Reader, ... Read more
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TOPAZ Trial Results on Muscle Therapy Likely by June, Scholar Rock Says
Efficacy and safety results from the TOPAZ Phase 2 trial evaluating apitegromab, a muscle-directed therapy for spinal muscular atrophy (SMA) being developed by Scholar Rock, ... Read more -
Advice From a Life Coach: Resolutions, Mindsets, and Emotions
Last summer, I met a new friend, Amber Bosselman, at the Virtual Cure SMA Conference. Amber is a certified life coach with a bachelor’s ... Read more -
Online for 2021, Team Cure SMA Race Series Planning Variety of Events
The COVID-19 pandemic has forced many activities to be online only, but in the case of this year’s Team Cure SMA endurance race series, ... Read more
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Researchers Discover Promising New Gene Therapy Approach for Spinal Muscular Atrophy
News
A new genetic approach to restore the expression of the gene that causes spinal muscular atrophy (SMA) in patients’ cells holds promise to effectively and permanently treat the disease, researchers suggest. The study, “Seamless genetic conversion of SMN2 to ... Read more