Welcome to ‘Wandering the Lines,’ Our Journey Into the Unknown
“I wish I were normal,” I used to say to my mum. I told her this at other kids’ birthday parties, when they played ... Read more
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Better Ways of Capturing Progression in Types 2 and 3 Identified in Study
Four measures of motor ability — including one of continuous upper limb activity that can be done at the home — reliably detect significant ... Read more -
Being My Own Health Advocate Kindles Multidimensional Healing
When I say, “I don’t need my SMA to be fixed,” I mean it from the bottom of my heart. Last month, I set ... Read more -
NORD’s Caregiver Respite Program Continues Through Pandemic
Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet ... Read more -
Cure SMA Booklet Outlines Possible Risks, Benefits of Combining Treatments
A free resource booklet, published by Cure SMA, may help people with spinal muscular atrophy (SMA) to better understand the possible risks and benefits of ... 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