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108. Choroid plexus mis-splicing and altered cerebrospinal fluid composition in myotonic dystrophy type 1

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

110. Assessing Bulbar Function in Spinal Muscular Atrophy Using Patient-Reported Outcomes

111. Efficacy and Safety of Avalglucosidase Alfa in Patients with Late-Onset Pompe Disease after 97 Weeks:A Phase 3 Randomized Clinical Trial

112. 2-Year Change in Revised Hammersmith Scale Scores in a Large Cohort of Untreated Paediatric Type 2 and 3 SMA Participants

113. Myostatin and follistatin as monitoring and prognostic biomarkers in dysferlinopathy

114. Expanding the muscle imaging spectrum in dysferlinopathy: description of an outlier population from the classical MRI pattern

117. Assessment of disease progression in dysferlinopathy: A 1-year cohort study

119. 2-Year Change in Revised Hammersmith Scale Scores in a Large Cohort of Untreated Paediatric Type 2 and 3 SMA Participants

120. Efficacy and safety of avalglucosidase alfa in participants with late-onset Pompe disease after 145 weeks of treatment during the COMET trial

121. AT845 gene replacement therapy for late onset Pompe disease: An update on safety and preliminary efficacy data from FORTIS, a phase I/II open-label clinical study

122. O19: Integrated analyses of data from clinical trials of delandistrogene moxeparvovec gene therapy in DMD*

125. Consensus-based care recommendations for adults with myotonic dystrophy type 1

127. Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial

132. Water T2 could predict functional decline in patients with dysferlinopathy

135. Quantitative Evaluation of Lower Extremity Joint Contractures in Spinal Muscular Atrophy: Implications for Motor Function

136. Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

146. Conservation and Use of Coastal Wetland Forests in Louisiana

148. Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial.

149. Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial

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