#MDA2022 – Newborns Treated With Evrysdi Standing, Walking 1 Year Later
In the RAINBOWFISH trial testing oral Evrysdi (risdiplam) in babies with spinal muscular atrophy (SMA) but no evident symptoms, most…
Articles by Marisa Wexler, MS
#MDA2022 – Data: Zolgensma Given After 6 Months Benefits SMA Babies
Even when given to young children with spinal muscular atrophy (SMA) at ages older than 6 months, the gene…
#MDA2022 – Evrysdi Safe at 1 Year in Previously Treated Patients
Treatment with Evrysdi (risdiplam) continues to be safe and well tolerated in people with spinal muscular atrophy (SMA) who…
#MDA2022 – Motor Benefits of Evrysdi for Children, Adults in SUNFISH Now at 3 Years
Children and young adults with spinal muscular atrophy (SMA) types 2 and 3 continue to report improved or stabilized…
#MDA2022 – ‘Amazing’ Walking Ability Seen in SMA Children on Spinraza
Most children with spinal muscular atrophy (SMA) who were presymptomatic and treated with Spinraza (nusinersen) in the NURTURE…
Newborn Screening for SMA Now Launched in Canadian Province
Babies born in the Canadian province of Saskatchewan will now be tested for spinal muscular atrophy (SMA) as part…
Autopsy Shows Little Brain Damage in Type 2 Patient Who Lived to 61
A man with spinal muscular atrophy (SMA) type 2 in Japan who lived until his 60s was found after…
Study Highlights Gaps in Knowledge Among Prenatal Genetic Counselors
Many prenatal genetic counselors are unfamiliar with important concepts related to spinal muscular atrophy (SMA) and its treatment, according…
Evrysdi for Infants Under 2 Months Old Given FDA Priority Review
The U.S. Food and Drug Administration (FDA) is giving priority review to a request that the oral therapy Evrysdi (risdiplam)…
Equity Concerns as Medicaid for Spinraza, Zolgensma Varies by State
Within the U.S., there is marked state-to-state variation in guidelines for whether Medicaid will cover Spinraza (nusinersen) or…