Results 201 to 210 of about 20,899 (290)

A Systematic Literature Review of the Natural History of Respiratory, Swallowing, Feeding, and Speech Functions in Spinal Muscular Atrophy (SMA). [PDF]

J Neuromuscul Dis
Martí Y, Aponte Ribero V, Batson S, Mitchell S, Gorni K, Gusset N, Oskoui M, Servais L, Deconinck N, McGrattan KE, Mercuri E, Sutherland CS.   +11 more
europepmc   +1 more source

HTA210 Effectiveness of Generic Quality of Life (QoL) Questionnaires in Spinal Muscular Atrophy (SMA) and Duchenne Muscular Dystrophy (DMD) [PDF]

M Larkova, M Siekerman, Susanne Winther Olsen, Andersen Ke, Magne Thoresen   +4 more
openalex   +1 more source

Evaluating Benefit-risk Decision-making in Spinal Muscular Atrophy: A First-ever Study to Assess Risk Tolerance in the SMA Patient Community [PDF]

, 2019
Rosángel Cruz, Lisa Belter, Mike Wasnock, Al Nazarelli, Jill Jarecki   +4 more
openalex   +1 more source

SMA CARNI-VAL TRIAL PART II: A Prospective, Single-Armed Trial of L-Carnitine and Valproic Acid in Ambulatory Children with Spinal Muscular Atrophy

, 2011
John T. Kissel, Charles Scott, Sandra P. Reyna, Thomas O. Crawford, Louise R. Simard, Kristin J. Krosschell, Gyula Acsádi, Bakri Elsheik, Mary Schroth, Guy D’Anjou, Bernard LaSalle, Thomas W. Prior, Susan Sorenson, Jo Anne Maczulski, Mark B. Bromberg, Gary M. Chan, Kathryn J. Swoboda, for the Project Cure Spinal Muscular Atrophy Investigators' Network   +17 more
openalex   +2 more sources

Quality of life data for individuals affected by Spinal Muscular Atrophy: A baseline dataset from the Cure SMA Community Update Survey [PDF]

, 2020
Lisa Belter, Rosángel Cruz, Jill Jarecki   +2 more
openalex   +1 more source

Assessment of Barriers to Referral and Appointment Wait Times for the Evaluation of Spinal Muscular Atrophy (SMA): Findings from a Web-Based Physician Survey. [PDF]

Neurol Ther
Curry MA, Cruz RE, Belter LT, Schroth MK, Jarecki J.   +4 more
europepmc   +1 more source

Pharmacological and clinical profile of Onasemnogene Aveparvovec, the first gene therapy for spinal muscular atrophy (SMA)

, 2021
Ayaka Atsumi, Yoneda Tomohiro, Ken Tsuchida, Yusuke Kagawa, Shunsuke Tominaga, Kazuho Kawase, Nobutaka Kikuchi   +6 more
openalex   +2 more sources

Development and validation of a 4-color multiplexing spinal muscular atrophy (SMA) genotyping assay on a novel integrated digital PCR instrument. [PDF]

Sci Rep, 2020
Jiang L, Lin R, Gallagher S, Zayac A, Butchbach MER, Hung P.   +5 more
europepmc   +1 more source

Peripheral drug delivery for spinal muscular atrophy (SMA) [PDF]

Science-Business eXchange, 2011
openaire   +1 more source

Spinal Muscular Atrophy (SMA) Subtype Concordance in Siblings: Findings From the Cure SMA Cohort. [PDF]

J Neuromuscul Dis, 2020
Jones CC, Cook SF, Jarecki J, Belter L, Reyna SP, Staropoli J, Farwell W, Hobby K.   +7 more
europepmc   +1 more source