Unexpected Outcomes In A Child with SMA type 1 (Pre-Spinraza treatment)
April 23, 2018
Background: Spinal muscular atrophy is an autosomal recessive genetic disorder. Type I (Werdnig-Hoffman, acute) is the most severe form of SMA. Onset is noted at 0-3 months due to hypotonia and severe weakness, feeding difficulties and/or need for respiratory support. … Read more