228 results on '"Case, Laura E."'
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2. Phase I study of liver depot gene therapy in late-onset Pompe disease
3. Diagnosis and management of glycogen storage disease type IV, including adult polyglucosan body disease: A clinical practice resource
4. IOPD entering adulthood: Lessons from two decades of ERT experience
5. Real-world outcomes from a series of patients with late onset Pompe disease who switched from alglucosidase alfa to avalglucosidase alfa
6. Higher dosing of alglucosidase alfa improves outcomes in children with Pompe disease: a clinical study and review of the literature
7. Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management
8. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management
9. Screening data from 19 patients with late‐onset Pompe disease for a phase I clinical trial of AAV8 vector‐mediated gene therapy
10. Physical Therapy and Orthotic Devices
11. Commentary on “Progression of Ankle Plantarflexion Contractures and Functional Decline in Duchenne Muscular Dystrophy: Implications for Physical Therapy Management”
12. Physical Activity Levels of Children With Down Syndrome
13. Screening data from 19 patients with late‐onset Pompe disease for a phase I clinical trial of AAV8 vector‐mediated gene therapy.
14. Safety and efficacy of alternative alglucosidase alfa regimens in Pompe disease
15. Effect of Autologous Cord Blood Infusion on Motor Function and Brain Connectivity in Young Children with Cerebral Palsy: A Randomized, Placebo‐Controlled Trial
16. Optimizing clinical outcomes: The journey of twins with CRIM-negative infantile-onset Pompe disease on high-dose enzyme replacement therapy and immunomodulation
17. Motor function and safety after allogeneic cord blood and cord tissue‐derived mesenchymal stromal cells in cerebral palsy: An open‐label, randomized trial
18. Interventions for Gait Training in Children With Spinal Cord Impairments: A Scoping Review
19. A RETROSPECTIVE LONGITUDINAL STUDY AND COMPREHENSIVE REVIEW OF ADULT PATIENTS WITH GLYCOGEN STORAGE DISEASE TYPE III
20. Early clinical phenotype of late onset Pompe disease: Lessons learned from newborn screening
21. A retrospective longitudinal study and comprehensive review of adult patients with glycogen storage disease type III
22. Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care
23. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management
24. Physical therapy assessment and whole-body magnetic resonance imaging findings in children with glycogen storage disease type IIIa: A clinical study and review of the literature
25. Correlation between quantitative whole-body muscle magnetic resonance imaging and clinical muscle weakness in pompe disease
26. Sibling Umbilical Cord Blood Infusion is Safe in Young Children with Cerebral Palsy
27. Oropharyngeal Dysphagia in Infants and Children with Infantile Pompe Disease
28. Fractures in children with Pompe disease: a potentiallong-term complication
29. Generating color-coded anatomic muscle maps for correlation of quantitative magnetic resonance imaging analysis with clinical examination in neuromuscular disorders
30. EXERCISE AND DUCHENNE MUSCULAR DYSTROPHY: WHERE WE HAVE BEEN AND WHERE WE NEED TO GO
31. Infantile Pompe disease on ERT—Update on clinical presentation, musculoskeletal management, and exercise considerations
32. Training, detraining, and retraining: Two 12-week respiratory muscle training regimens in a child with infantile-onset Pompe disease
33. Improved muscle function in a phase I/II clinical trial of albuterol in Pompe disease
34. Higher dosing of alglucosidase alfa improves outcomes in children with Pompe disease
35. A phase 1 study of gene therapy with ACTUS-101 in late-onset Pompe disease
36. Early-onset of symptoms and clinical course of Pompe disease associated with the c.-32–13 T > G variant
37. A Transition Toolkit for Duchenne Muscular Dystrophy
38. Rehabilitation Management of the Patient With Duchenne Muscular Dystrophy
39. Correction of Biochemical Abnormalities and Improved Muscle Function in a Phase I/II Clinical Trial of Clenbuterol in Pompe Disease
40. Insight into the phenotype of infants with Pompe disease identified by newborn screening with the common c.-32-13T > G “late-onset” GAA variant
41. The emerging phenotype of late-onset Pompe disease: A systematic literature review
42. Alglucosidase alfa enzyme replacement therapy as a therapeutic approach for a patient presenting with a PRKAG2 mutation
43. Commentary on “Supported Standing in Boys With Duchenne Muscular Dystrophy”
44. Physical therapy management of infants and children with hypophosphatasia
45. Respiratory muscle training (RMT) in late-onset Pompe disease (LOPD): Effects of training and detraining
46. Characterization of gait in late onset Pompe disease
47. Exome sequencing results in successful riboflavin treatment of a rapidly progressive neurological condition
48. Sustained therapeutic response to riboflavin in a child with a progressive neurological condition, diagnosed by whole-exome sequencing
49. Commentary on “Cardiopulmonary Exercise Testing in Children and Adolescents With Dystrophinopathies
50. [Commentary on] Interventions for Gait Training in Children With Spinal Cord Impairments: A Scoping Review.
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