Author Archives: Jose Marques Lopes PhD

#AANAM — Experts Discuss Improved Survival, Key Motor Milestones Achieved with Risdiplam in SMA Trials

Treatment with the experimental oral therapy risdiplam has been enabling patients with spinal muscular atrophy (SMA) to achieve key development milestones and clinically meaningful motor improvements beyond the natural history of the disease. Susan Begelman, MD, vice president, Neurology, Nonmalignant Hematology, Influenza, U.S. Medical Affairs at Genentech,…

#AANAM – Spinraza Showing Long-term Benefits in All SMA Types, Biogen Says in Interview

Long-term results show Spinraza (nusinersen) is of unprecedented benefit to a broad range of spinal muscular atrophy (SMA) patients, said Wildon Farwell, executive director of clinical development at Biogen, which markets this approved treatment. With clinical development that spans “presymptomatic infants, to symptomatic infants, to symptomatic children,” Spinraza has “demonstrated…