Results 121 to 130 of about 2,038 (184)

A Chemically Induced CRISPR/dCas13^FCPF Platform for Precise and Programmable RNA Regulation. [PDF]

J Med Chem
Hasselbeck S, Wang J, Bai Z, Hüfner T, Hummer G, Grote P, Cheng X.   +6 more
europepmc   +1 more source

Efficacy and safety of risdiplam in adults with 5q-associated spinal muscular atrophy: a nationwide observational cohort study in Austria. [PDF]

EClinicalMedicine
Keritam O, Erdler M, Fasching B, Zulehner G, Rath J, Krenn M, Waldhör T, Gruber VA, Langweil N, Kiss C, Griedl TA, Gold V, Wanschitz J, Hotter A, Kleinveld VEA, Horlings CGC, Erber A, Schernhammer E, Troger J, Grinzinger S, Müller P, Langenscheidt D, Rappold M, Wiesenhofer A, Gosk-Tomek M, Knipp F, Mahal S, Bernert G, Baumann M, Zimprich F, Topakian R, Eggers C, Quasthoff S, Löscher W, Cetin H.   +34 more
europepmc   +1 more source

Swallowing and feeding after disease-modifying treatment for spinal muscular atrophy: a systematic review of assessment modalities and outcomes. [PDF]

Orphanet J Rare Dis
Martí Y, Gorni K, Kumari S, Mahajan A, Baranello G, Waele L, McGrattan KE.   +6 more
europepmc   +1 more source

Drug Screening and Drug Repositioning as Promising Therapeutic Approaches for Spinal Muscular Atrophy Treatment [PDF]

, 2020
Boido, Marina Maria, Daniela Maria Rasà, Menduti, Giovanna, Stanga, Serena   +3 more
core   +1 more source

Analyse der gesundheitsbezogenen Lebensqualität von Patienten mit spinaler Muskelatrophie (SMA) unter der Therapie mit Nusinersen und Ermittlung der Belastung ihrer pflegenden Angehörigen [PDF]

Velling, Ann-Kathrin
core   +1 more source

Newborn Screening for Spinal Muscular Atrophy in the UK: Use of Modelling to Identify Priorities for Ongoing Evaluation. [PDF]

Int J Neonatal Screen
Thokala P, Bessey A, Knowles R, Marshall J, Visintin C, Lawton M, Lombardo S.   +6 more
europepmc   +1 more source

Five-year disease-modifying therapeutic experience of 102 Chinese paediatric 5q-spinal muscular atrophy: a retrospective analysis. [PDF]

Brain Commun
Jiang M, Liang C, Zhang Y, Zheng K, Xu K, He L, Tao J, Wu W, Zheng R, Rao M, Zhang W, Zhou W, Liu L.   +12 more
europepmc   +1 more source

Pregnancy in women with spinal muscular atrophy (SMA): maternal and neonatal outcomes with multi-speciality management. [PDF]

J Neurol
Cohen D, Nana M, Hart N, Arrandale L, Kelleher C, Nelson-Piercy C.   +5 more
europepmc   +1 more source

Disease characteristics and treatment status of genetically confirmed spinal muscular atrophy patients: a cross-sectional survey in China. [PDF]

BMC Neurol
Zhang D, Yao F, Ling C, Jiang S, Yang X, Dai Y.   +5 more
europepmc   +1 more source