July 19th 2024
SMA, a rare genetic neurodegenerative disorder affecting approximately 1 in 10,000 newborns, is typically fatal for untreated children by age two. However, the approval of disease-modifying medications have transformed the treatment landscape.
December 16th 2021
Study Looks At Novel Administration of Spinraza
October 14th 2021A new method for delivering this treatment drug for spinal muscular atrophy offers an option for patients with advanced cases of the disease. But investigators found that the new method may also carry risks of mechanical failure and infection.
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Spinal Muscular Atrophy Drug Risdiplam Proves Success in Infants
March 2nd 2021Patients treated with risdiplam at 12 months demonstrated significant improvement in survival and developmental milestones, with 19 of 21 (90%) infants able to survive without permanent ventilation and 7 (41%) infants able to sit without support for at least 5 seconds.
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