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Carolina Tesi Rocha, MD

Articles

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.