13 results on '"Day, John"'
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2. Disease Trajectories in the Revised Hammersmith Scale in a Cohort of Untreated Patients with Spinal Muscular Atrophy types 2 and 3
3. Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)
4. DEVOTE Study Exploring Higher Dose of Nusinersen in Spinal Muscular Atrophy: Study Design and Part A Results.
5. Assessing Bulbar Function in Spinal Muscular Atrophy Using Patient-Reported Outcomes
6. Assessing Bulbar Function in Spinal Muscular Atrophy Using Patient-Reported Outcomes
7. Motor Responses in Pediatric Pompe Disease in the ADVANCE Participant Cohort.
8. RESTORE: A Prospective Multinational Registry of Patients with Genetically Confirmed Spinal Muscular Atrophy - Rationale and Study Design
9. Revised Recommendations for the Treatment of Infants Diagnosed with Spinal Muscular Atrophy Via Newborn Screening Who Have 4 Copies of SMN2
10. Perspectives on Spinraza (Nusinersen) Treatment Study: Views of Individuals and Parents of Children Diagnosed with Spinal Muscular Atrophy
11. Treatment Algorithm for Infants Diagnosed with Spinal Muscular Atrophy through Newborn Screening
12. Evaluator Training and Reliability for SMA Global Nusinersen Trials1
13. Modifier Gene Candidates in Charcot-Marie-Tooth Disease Type 1A: A Case-Only Genome-Wide Association Study
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