Works matching IS 22143599 AND DT 2022 AND VI 9 AND IP 6

Results: 11

California's experience with SMA newborn screening: A successful path to early intervention.

Published in:

Journal of Neuromuscular Diseases, 2022, v. 9, n. 6, p. 777, doi. 10.3233/JND-221561

By:

Matteson, Jamie;
Wu, Cindy H.;
Mathur, Deepika;
Tang, Hao;
Sciortino, Stanley;
Feuchtbaum, Lisa;
Bishop, Tracey;
Sharma, Sudhir C.;
Neogi, Partha;
Fitzgibbon, Ina;
Olney, Richard S.

Publication type:

Article

Gene Therapy for Neuromuscular Diseases: Health Economic Challenges and Future Perspectives.

Published in:

Journal of Neuromuscular Diseases, 2022, v. 9, n. 6, p. 675, doi. 10.3233/JND-221540

By:

Landfeldt, Erik

Publication type:

Article

Clinical Trial Readiness for Spinal Muscular Atrophy: Experience of an International Educational-Training Initiative.

Published in:

Journal of Neuromuscular Diseases, 2022, v. 9, n. 6, p. 809, doi. 10.3233/JND-221538

By:

Tizzano, Eduardo F.;
Christie-Brown, Vanessa;
Baranello, Giovanni;
Germanenko, Olga;
Gray, Allyson;
Krstic, Marija;
Lilien, Charlotte;
Patel, Hinal;
Servais, Laurent;
Scoto, Mariacristina

Publication type:

Article

Gene Therapy for Duchenne Muscular Dystrophy: Unlocking the Opportunities in Countries in the Middle East and Beyond.

Published in:

Journal of Neuromuscular Diseases, 2022, v. 9, n. 6, p. 787, doi. 10.3233/JND-221528

By:

Elbashir, Haitham;
Fathalla, Waseem;
Mundada, Vivek;
Iqbal, Mehtab;
Al Tawari, Asmaa A;
Chandratre, Saleel;
Bastaki, Laila;
Romany, Ingy;
Ismayl, Omar;
Abou Tayoun, Ahmad

Publication type:

Article

Perioperative Care for Patients with Neuromuscular Disorders in the Netherlands – A Questionnaire Study Among Anaesthesiologists, Neurologists and Clinical Geneticists.

Published in:

Journal of Neuromuscular Diseases, 2022, v. 9, n. 6, p. 765, doi. 10.3233/JND-221512

By:

van den Bersselaar, L.R.;
Gubbels, M.H.M.;
Jungbluth, H.;
Schouten, M.I.;
van der Kooi, A.J.;
Quinlivan, R.;
Scheffer, G.J.;
Riazi, S.;
Snoeck, M.M.J.;
Voermans, N.C.

Publication type:

Article

Associations Between Self-Reported Behavioral and Learning Concerns and DMD Isoforms in Duchenne Muscular Dystrophy.

Published in:

Journal of Neuromuscular Diseases, 2022, v. 9, n. 6, p. 757, doi. 10.3233/JND-220821

By:

Counterman, Kevin J.;
Fatovic, Kathy;
Good, Daniel C.;
Martin, Ann S.;
Dasgupta, Sonali;
Anziska, Yaacov

Publication type:

Article

Cataract, abnormal electroretinogram and visual evoked potentials in a child with SMA-LED2 - extending the phenotype.

Published in:

Journal of Neuromuscular Diseases, 2022, v. 9, n. 6, p. 803, doi. 10.3233/JND-220818

By:

Oliwa, Agata;
Joseph, Shuko;
Millar, Eoghan;
Horrocks, Iain;
Penman, Dawn;
Baptista, Julia;
Cullup, Thomas;
Constantinou, Panayiotis;
Heuchan, Anne-Marie;
Hamilton, Ruth;
Longman, Cheryl

Publication type:

Article

Characterizing the Occurrence of Key Clinical Milestones in Duchenne Muscular Dystrophy in the United States Using Real-World Data.

Published in:

Journal of Neuromuscular Diseases, 2022, v. 9, n. 6, p. 689, doi. 10.3233/JND-220816

By:

Szabo, Shelagh M.;
Klimchak, Alexa C.;
Qian, Christina;
Iannaccone, Susan;
Popoff, Evan;
Gooch, Katherine L.

Publication type:

Article

Motor Responses in Pediatric Pompe Disease in the ADVANCE Participant Cohort.

Published in:

Journal of Neuromuscular Diseases, 2022, v. 9, n. 6, p. 713, doi. 10.3233/JND-210784

By:

Duong, Tina;
Kishnani, Priya S.;
An Haack, Kristina;
Foster, Meredith C.;
Gibson, James B.;
Wilson, Catherine;
Hahn, Si Houn;
Hillman, Richard;
Kronn, David;
Leslie, Nancy D.;
Peña, Loren D.M.;
Sparks, Susan E.;
Stockton, David W.;
Tanpaiboon, Pranoot;
Day, John W.

Publication type:

Article

The Relation Between Nutritional Intake and Body Weight in 4–18 Year Old Patients with DMD: What could possibly be done to prevent weight gain?

Published in:

Journal of Neuromuscular Diseases, 2022, v. 9, n. 6, p. 701, doi. 10.3233/JND-220796

By:

Dietvorst, Carmen A.W.;
Bot, Daphne;
van der Holst, Menno;
Niks, Erik H.

Publication type:

Article

Multiple Case Study of Changes in Participation of Adults with Myotonic Dystrophy Type 1: Importance of Redesigning Accomplishment and Resilience.

Published in:

Journal of Neuromuscular Diseases, 2022, v. 9, n. 6, p. 731, doi. 10.3233/JND-210780

By:

Raymond, Kateri;
Gagnon, Cynthia;
Levasseur, Mélanie

Publication type:

Article