This week's must-know community updates, latest research & events
March 13, 2025 • Estimated Reading Time: 1 minute
New This Week
Exciting news! There is now a new section featuring clinical trials that are actively recruiting patients. This highly requested addition gives you direct access to cutting-edge research opportunities that could shape the future of treatment.
Stay informed about potentially life-changing studies and let us know what you think by replying to this email!
A recent study by O'Leary (2025) has shown promising results for treating spinal muscular atrophy (SMA) before birth. The study, which is an N-of-1 trial, meaning it focused on a single patient, found that the drug risdiplam can pass from the mother to the fetus through the placenta. This is significant because it suggests that prenatal treatment could potentially prevent the onset of SMA symptoms before a child is born. The implications of this study are substantial, as early intervention is crucial for SMA, a disease where early symptom onset can lead to more severe outcomes. The ability of risdiplam to cross the placental barrier opens up the possibility of treating the disease at a stage when it could be most beneficial, potentially improving the quality of life for those affected by SMA.
Clinical Trials
This is a list of upcoming or ongoing clinical trials that are actively recruiting and have been listed or updated in the last two weeks:
More Info 
Add to Calendar