Unmet Needs in the Treatment of Spinal Muscular Atrophy
A panel of experts considers current unmet needs in SMA by emphasizing the need for further study of combination therapy, more comprehensive newborn screening, and more effective early diagnosis and treatment.
The JEWELFISH Trial of Risdiplam in Pretreated SMA
Key opinion leader in neurology Claudia Chiriboga, MD, MPH provides insight into response to risdiplam in patients with SMA who were not naïve to treatment as studied in the JEWELFISH clinical trial.
The SUNFISH Trial of Risdiplam in Type 2 and Type 3 SMA
Tom Crawford, MD, examines data from the SUNFISH trial of patients with type 2 and type 3 SMA and emphasizes the need for further data before risdiplam can be directly compared to other treatment options.
The FIREFISH Trial of Risdiplam in Type 1 SMA
Basil Darras, MD discusses the FIREFISH trial of risdiplam in infants with type 1 symptomatic SMA and comments on the difficulty of comparing agents when trials have had different enrollment criteria.