Spinal muscular atrophy (SMA) is a progressive genetic neuromuscular ... In this report, we present the case discovery of a four-month-old male patient with SMA type 1, presenting with generalized ...
So in type 1, which mainly affects the infants or the little babies, and so those little babies are usually symptomatic or present with weakness fairly early on in their lifetime within the first ...
Mid-Day on MSN9d
Two-year-old has only 15 days of medicines leftThe parents of two-year-old Aakriti Singh diagnosed with Spinal Muscular Atrophy – SMA type-1, are worried about her next ...
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Thirteen of 22 (59.1%) patients treated with onasemnogene abeparvovec achieved the milestone of functional independent sitting for ≥30 seconds at the 18-month visit (P<0.0001 vs PNCR). Twenty-one of ...
More research is needed to confirm gray matter, the darker tissue found in the brain and spinal cord, “as a potential disease ...
Application is supported by results from the DEVOTE study, which suggested that two doses of Spinraza (nusinerse) 50 mg taken ...
cbc.ca on MSN8d
Man with degenerative disease pleads for Manitoba to cover treatment while it asks drug agency to reviewA man with a muscle-wasting disease is pleased Manitoba is asking a federal agency to reconsider its opposition to a ...
These and other socioeconomic characteristics are shown in Table 1. PedsQL™ 4.0 – patient self-assessment (n=49) and carer assessment (n=115) by spinal muscular atrophy type. The lowest and highest ...
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