Results 1 to 10 of about 20,899 (290)

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   +3 more sources

SMA-MAP: A Plasma Protein Panel for Spinal Muscular Atrophy

PLoS ONE, 2013
Spinal Muscular Atrophy (SMA) presents challenges in (i) monitoring disease activity and predicting progression, (ii) designing trials that allow rapid assessment of candidate therapies, and (iii) understanding molecular causes and consequences of the disease.
Dione T Kobayashi, Jing Shi, Laurie Stephen, Karri L Ballard, Ruth Dewey, James Mapes, Brett Chung, Kathleen McCarthy, Kathryn J Swoboda, Thomas O Crawford, Rebecca Li, Thomas Plasterer, Cynthia Joyce, Biomarkers for Spinal Muscular Atrophy Study Group, Wendy K Chung, Petra Kaufmann, Basil T Darras, Richard S Finkel, Douglas M Sproule, William B Martens, Michael P McDermott, Darryl C De Vivo, Pediatric Neuromuscular Clinical Research Network, Michael G Walker, Karen S Chen   +24 more
openaire   +7 more sources

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   +3 more sources

A transcriptomics-based drug repositioning approach to identify drugs with similar activities for the treatment of muscle pathologies in spinal muscular atrophy (SMA) models [PDF]

, 2023
Hoolachan JM, McCallion E, Sutton ER, Çetin Ö, Pacheco-Torres P, Dimitriadi M, Okoh M, Walter LM, Claus P, Wood MJ, Tonge DP, Bowerman M.   +11 more
europepmc   +2 more sources

Awareness screening and referral patterns among pediatricians in the United States related to early clinical features of spinal muscular atrophy (SMA). [PDF]

BMC Pediatr, 2021
Curry M, Cruz R, Belter L, Schroth M, Lenz M, Jarecki J.   +5 more
europepmc   +3 more sources

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   +3 more sources

Correction to: Systematic Literature Review to Assess Economic Evaluations in Spinal Muscular Atrophy (SMA). [PDF]

Pharmacoeconomics, 2022
Paracha N, Hudson P, Mitchell S, Sutherland CS.   +3 more
europepmc   +3 more sources

Activation of Muscle-Specific Kinase (MuSK) Reduces Neuromuscular Defects in the Delta7 Mouse Model of Spinal Muscular Atrophy (SMA). [PDF]

Int J Mol Sci, 2021
Feng Z, Lam S, Tenn ES, Ghosh AS, Cantor S, Zhang W, Yen PF, Chen KS, Burden S, Paushkin S, Ayalon G, Ko CP.   +11 more
europepmc   +3 more sources

Systematic Literature Review to Assess Economic Evaluations in Spinal Muscular Atrophy (SMA). [PDF]

Pharmacoeconomics, 2022
Paracha N, Hudson P, Mitchell S, Sutherland CS.   +3 more
europepmc   +3 more sources

Design and Validation of a Clinical Outcome Measure for Adolescents and Adult Patients with Spinal Muscular Atrophy: SMA Life Study Protocol. [PDF]

Neurol Ther
Rebollo P, García-López S, Povedano M, Cattinari MG, Martínez-Moreno M, Terrancle Á, Cabello-Moruno R, Vázquez-Costa JF.   +7 more
europepmc   +3 more sources