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Continued benefit of nusinersen initiated in the presymptomatic stage of spinal muscular atrophy: 5-year update of the NURTURE study.

Thomas O. Crawford, Aledie Navas Nazario, Angela D. Paradis, Astrid Pechmann, Cong Zhu, Darryl C. De Vivo, Enrico Bertini, Francy Shu, Haluk Topaloglu, Janbernd Kirschner, Julie A. Parsons, Kathryn J. Swoboda, Monique M. Ryan, Nancy L. Kuntz, Richard Foster, Richard S. Finkel, Russell Chin, Russell J. Butterfield, Stephanie Raynaud, Tawfeg Ben-Omran, Tiffany R. Lago, Valeria A. Sansone, Wuh-Liang Hwu, Yuh-Jyh Jong, Zdenek Berger

Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)

Longitudinal Assessment of Timed Function Tests in Ambulatory Individuals with SMA Treated with Nusinersen

Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG).

Finkel, Richard S; Darras, Basil T; Mendell, Jerry R; Day, John W; Kuntz, Nancy L; Connolly, Anne M; Zaidman, Craig M; Crawford, Thomas O; Butterfield, Russell J; Shieh, Perry B; Tennekoon, Gihan; Brandsema, John F; Iannaccone, Susan T; Shoffner, John; Kavanagh, Sarah; Macek, Thomas A; Tauscher-Wisniewski, Sitra

Longitudinal Assessment of Timed Function Tests Over Time in Ambulatory Individuals with SMA Treated with Nusinersen.

Krosschell, Kristin J; Brown, Laurey; Hoffman, Katie; Zumpf, Katelyn B; Munson, Hannah; Bidwell, Jessa; Schulte, Duncan P; Schwaede, Abigail N; Buehner, Amber N; DiDonato, Christine J; Kuntz, Nancy L; Rao, Vamshi K

A Virtual Reality Exergame: Clinician-Guided Breathing and Relaxation for Children with Muscular Dystrophy

Updates in the Use of Vamorolone and Steroids in the Treatment of Duchenne Muscular Dystrophy

Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.

Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy

INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular Myopathy

An expanded access program of risdiplam for patients with Type 1 or 2 spinal muscular atrophy.

An expanded access program of risdiplam for patients with Type 1 or 2 spinal muscular atrophy