Search

Search Constraints

You searched for: Author/Creator Schultz, Meredith

Search Results

1. 014 AVXS-101 gene-replacement therapy (GRT) in presymptomatic spinal muscular atrophy (SMA): study update. (29th July 2019)

2. 15.33 AVXS-101 in presymptomatic spinal muscular atrophy (SMA). Issue 12 (14th November 2019)

3. 250 AVXS-101 phase 3 study in spinal muscular atrophy type 1. Issue 12 (14th November 2019)

4. A Prospective, Crossover Survey Study of Child- and Proxy-Reported Quality of Life According to Spinal Muscular Atrophy Type and Medical Interventions. (April 2020)

5. Digital endpoints for self‐administered home‐based functional assessment in pediatric Friedreich's ataxia. Issue 9 (6th August 2021)

6. High Incidence of Noonan Syndrome Features Including Short Stature and Pulmonic Stenosis in Patients carrying NF1 Missense Mutations Affecting p.Arg1809: Genotype–Phenotype Correlation. Issue 11 (21st August 2015)

7. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial. Issue 4 (April 2021)