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Basil Darras, MD

Articles

Complexities With SMN2 Copies in Spinal Muscular Atrophy: Basil Darras, MD

August 01, 2022

The associate neurologist-in-chief at Boston Children’s Hospital discussed the 4-copy conundrum and whether treatment decisions differ based on SMN2 copies for patients with spinal muscular atrophy. [WATCH TIME: 2 minutes]

Unmet Needs in the Treatment of Spinal Muscular Atrophy

June 11, 2021

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 FIREFISH Trial of Risdiplam in Type 1 SMA

June 04, 2021

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.

The SPR1NT Trial of Onasemnogene Abeparvovec in SMA

May 27, 2021

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.