June 11, 2021
Video
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.
June 11, 2021
Video
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.
June 04, 2021
Video
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.
June 04, 2021
Video
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.
May 27, 2021
Video
Expert pediatric neurologist Carolina Tesi Rocha, MD gives insight into the mechanism of action of risdiplam and comments on the need for further monitoring of its potential adverse effects.
May 27, 2021
Video
Claudia Chiriboga, MD, MPH reviews data from the SPR1NT clinical trial of patients with presymptomatic SMA and emphasizes how the trial highlights the importance of prenatal screening and early treatment.
May 21, 2021
Video
Basil Darras, MD provides an overview of safety and efficacy data from the phase 3 STR1VE clinical trial of onasemnogene abeparvovec in patients with symptomatic SMA up to 6 months of age.
May 21, 2021
Video
Tom Crawford, MD provides key insights into the START clinical trial of onasemnogene abeparvovec in symptomatic SMA and discusses its safety and efficacy for children up to 24 months of age.
May 14, 2021
Video
Key opinion leader in neurology Carolina Tesi Rocha, MD provides an overview of onasemnogene abeparvovec by discussing its mechanism of action, safety, and efficacy for patients with SMA.
May 14, 2021
Video
Experts in neurology Tom Crawford, MD and Claudia Chiriboga, MD, MPH discuss the impact of the NURTURE trial of nusinersen in patients with presymptomatic SMA.
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