| no |
title |
author |
magazine |
year |
volume |
issue |
page(s) |
type |
| 1 |
Associations Between Self-Reported Behavioral and Learning Concerns and DMD Isoforms in Duchenne Muscular Dystrophy
|
Counterman, Kevin J.
|
|
|
9 |
6 |
p. 757-764
|
article
|
| 2 |
California’s experience with SMA newborn screening: A successful path to early intervention
|
Matteson, Jamie
|
|
|
9 |
6 |
p. 777-785
|
article
|
| 3 |
Cataract, abnormal electroretinogram and visual evoked potentials in a child with SMA-LED2 - extending the phenotype
|
Oliwa, Agata
|
|
|
9 |
6 |
p. 803-808
|
article
|
| 4 |
Characterizing the Occurrence of Key Clinical Milestones in Duchenne Muscular Dystrophy in the United States Using Real-World Data
|
Szabo, Shelagh M.
|
|
|
9 |
6 |
p. 689-699
|
article
|
| 5 |
Clinical Trial Readiness for Spinal Muscular Atrophy: Experience of an International Educational-Training Initiative
|
Tizzano, Eduardo F.
|
|
|
9 |
6 |
p. 809-820
|
article
|
| 6 |
Gene Therapy for Duchenne Muscular Dystrophy: Unlocking the Opportunities in Countries in the Middle East and Beyond
|
Elbashir, Haitham
|
|
|
9 |
6 |
p. 787-801
|
article
|
| 7 |
Gene Therapy for Neuromuscular Diseases: Health Economic Challenges and Future Perspectives
|
Landfeldt, Erik
|
|
|
9 |
6 |
p. 675-688
|
article
|
| 8 |
Motor Responses in Pediatric Pompe Disease in the ADVANCE Participant Cohort
|
Duong, Tina
|
|
|
9 |
6 |
p. 713-730
|
article
|
| 9 |
Multiple Case Study of Changes in Participation of Adults with Myotonic Dystrophy Type 1: Importance of Redesigning Accomplishment and Resilience
|
Raymond, Kateri
|
|
|
9 |
6 |
p. 731-755
|
article
|
| 10 |
Perioperative Care for Patients with Neuromuscular Disorders in the Netherlands – A Questionnaire Study Among Anaesthesiologists, Neurologists and Clinical Geneticists
|
van den Bersselaar, L.R.
|
|
|
9 |
6 |
p. 765-775
|
article
|
| 11 |
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?
|
Dietvorst, Carmen A.W.
|
|
|
9 |
6 |
p. 701-711
|
article
|
Journal description