Results 121 to 130 of about 19,843 (235)

Emerging Gene Therapy Approaches in the Management of Spinal Muscular Atrophy (SMA): An Overview of Clinical Trials and Patent Landscape. [PDF]

Int J Mol Sci, 2023
Ponomarev AS, Chulpanova DS, Yanygina LM, Solovyeva VV, Rizvanov AA.   +4 more
europepmc   +1 more source

Gene Therapy for Spinal Muscular Atrophy (SMA): A Review of Current Challenges and Safety Considerations for Onasemnogene Abeparvovec (Zolgensma). [PDF]

Cureus, 2023
Ogbonmide T, Rathore R, Rangrej SB, Hutchinson S, Lewis M, Ojilere S, Carvalho V, Kelly I.   +7 more
europepmc   +1 more source

Correction to: Two‑year efficacy and safety of risdiplam in patients with type 2 or non‑ambulant type 3 spinal muscular atrophy (SMA). [PDF]

J Neurol, 2023
Oskoui M, Day JW, Deconinck N, Mazzone ES, Nascimento A, Saito K, Vuillerot C, Baranello G, Goemans N, Kirschner J, Kostera-Pruszczyk A, Servais L, Papp G, Gorni K, Kletzl H, Martin C, McIver T, Scalco RS, Staunton H, Yeung WY, Fontoura P, Mercuri E, SUNFISH Working Group.   +22 more
europepmc   +1 more source

General Anesthesia Plus Muscle Relaxant in a Patient with Kugelberg Welander Disease: A Case Report

Archives of Anesthesia and Critical Care, 2015
Spinal muscular atrophies (SMAs) represent a rare group of inherited disorders that cause progressive degeneration of the anterior horn cells of the spinal cord. The exact cause of the degeneration is unknown. Loss of these cells results in a progressive
Ebrahim Espahbodi, Amir Abbas Yaghooti, Hossein Sadrossadat, Mehrdad Shoroughi, Alireza Ebrahim Soltani, Mehrdad Goudarzi, Mehdi Sanatkar   +6 more
doaj  

Casting Light on Mechanical Properties of Lower and Upper Extremity Muscles in Children with Spinal Muscular Atrophy and Healthy Peers

Archives of Health Science and Research
Objective: This study aimed to compare the mechanical properties of upper and lower extremities between children with spinal muscular atrophy types 1 and 2 and healthy peers.
Seval KUTLUTÜRK-YIKILMAZ, Tülay ÇEVİK-SALDIRAN, Özgül ÖZTÜRK, Sedat ÖKTEM   +3 more
doaj   +1 more source

Parents' dilemma: A therapeutic decision for children with spinal muscular atrophy (SMA) type 1. [PDF]

Front Pediatr, 2022
Boursange S, Araneda M, Stalens C, Desguerre I, Barnerias C, Nougues MC, Isapof A, Quijano-Roy S, Blu Genestine N, Ouillade L, Martinez Jalilie M, Castiglioni C, Boespflug-Tanguy O, Gargiulo M, SMAPAR Study Group.   +14 more
europepmc   +1 more source

A call to introduce newborn screening for spinal muscular atrophy (SMA) in Scotland. [PDF]

Scott Med J, 2022
Gillingwater TH, McWilliam C, Horrocks I, McWilliam K, Hamilton M, Fletcher E, Williams N, Smith S, Parson SH.   +8 more
europepmc   +1 more source

Physiotherapy and rehabilitation in spinal muscular atrophy (SMA)

, 2021
No ...
openaire   +1 more source

The phospho-landscape of the survival of motoneuron protein (SMN) protein: relevance for spinal muscular atrophy (SMA). [PDF]

Cell Mol Life Sci, 2022
Detering NT, Schüning T, Hensel N, Claus P.   +3 more
europepmc   +1 more source

A Comprehensive Review on Spinal Muscular Atrophy (SMA) [PDF]

Perceptions in Reproductive Medicine, 2021
openaire   +1 more source