US, EU regulators to review higher-dose Spinraza for use in SMA
Regulators in the U.S. and the European Union have agreed to review Biogen’s applications seeking approval of a higher dose…
Articles by Margarida Maia, PhD
Gratitude programs may help with mental health, study finds
Gratitude and support from family and healthcare staff can help people with spinal muscular atrophy (SMA) feel more positive,…
Study calls for streamlined SMA treatment in Australia
Slightly more than half of spinal muscular atrophy (SMA) patients in Australia who gave feedback for a study on…
Apitegromab hits goal of boosting motor function in Phase 3 trial
Scholar Rock’s apitegromab led to motor function improvements over a year of treatment in young adults and children…
New SMA Europe documentary aims to raise awareness on research
SMA Europe has released a new documentary titled “One Community. Shared Dreams.” as part of an awareness initiative…
Newly identified mutation likely cause of SMARD1 in infant girl
A baby’s respiratory failure and need for mechanical ventilation were traced to spinal muscular atrophy with respiratory distress type…
SMA may involve changes that occur before birth, study suggests
Spinal muscular atrophy (SMA) may involve changes that occur before birth, during an embryo’s development in the womb, and…
Clinical benefit of apitegromab sustained for up to 3 years: Study
Three years of treatment with apitegromab, a muscle-targeting therapy being developed by Scholar Rock, continues to provide clinical…
Woman with unsteady walk is diagnosed with SMA in her late 40s
A woman with a long history of muscle weakness was found to have spinal muscular atrophy (SMA) in her…
Optoacoustic imaging may help monitor SMA progression in children
A method that uses laser light to get information from inside the body in a noninvasive way may help monitor…