Human milk boosts nutritional status of infant with SMA type 1
An intervention based on giving human milk instead of formula helped to improve the nutritional status of a baby with…
Articles by Marisa Wexler, MS
Long-term Spinraza found safe, effective for most adults with SMA
Long-term treatment with Spinraza (nusinersen) led to clinically relevant improvements in motor function for most adults and older children…
Newborn screening in Ukraine successfully launched amid war
Even though Ukraine has been at war for more than a year, a newborn screening program for spinal muscular…
Taldefgrobep alfa scores orphan drug designation in Europe
The European Commission has granted orphan drug status to taldefgrobep alfa as a potential treatment for spinal muscular atrophy…
Spinraza treatment found to benefit adults with SMA types 2, 3 in study
Treatment with Spinraza (nusinersen) can improve the movement ability of the diaphragm — the large muscle in the chest…
‘We are lucky and unlucky,’ SMA caregivers say of new treatments
For caregivers of children with spinal muscular atrophy (SMA), the availability of new disease-modifying treatments has offered hope —…
CHMP favors extending Evrysdi to newborns with SMA in the EU
The Committee for Medicinal Products for Human Use (CHMP) favors European Union (EU) approval of Evrysdi (risdiplam) being extended…
Levels of NfL protein in blood can pinpoint nerve damage side effects
Measuring levels of neurofilament light chain (NfL) in the blood could be used to detect subtle nerve damage that may…
Muscle contractures minor bother for SMA patients who can’t walk
Nonambulatory young adults with spinal muscular atrophy (SMA) who have contractures — when muscle and connective tissue around a…
Surveys show how SMA care was aided, hindered by pandemic
The COVID-19 pandemic made it more difficult for many people with spinal muscular atrophy (SMA) to access needed healthcare,…