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

Results: 11

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
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
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
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
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
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