760 results on '"Flanigan, Kevin"'
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2. Intron mutations and early transcription termination in Duchenne and Becker muscular dystrophy.
3. Literacy Intervention in the Middle Grades: Word Learning, Comprehension, and Strategy Instruction, Grades 4-8
4. Evaluation of safety and early efficacy of AAV gene therapy in mouse models of vanishing white matter disease
5. Molecular Genetic Therapies in the Muscular Dystrophies
6. Evaluating longitudinal therapy effects via the North Star Ambulatory Assessment.
7. Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model
8. The 'P' Word Revisited: 8 Principles for Tackling Today's Questions and Misconceptions about Phonics Instruction
9. CRISPR-Cas9 homology-independent targeted integration of exons 1–19 restores full-length dystrophin in mice
10. Word Study with Students Who Struggle: Reading, Vocabulary, and Spelling Instruction, Grades 4-12
11. Inherited myopathy plus: Double-trouble from rare neuromuscular disorders
12. Access to novel therapies for Duchenne muscular dystrophy—Insights from expert treating physicians
13. Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse
14. Assessment for Reading Instruction. Fourth Edition
15. Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy.
16. A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trial.
17. Draft Guidance for Industry Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and Related Dystrophinopathies – Developing Potential Treatments for the Entire Spectrum of Disease
18. Health related quality of life in young, steroid-naïve boys with Duchenne muscular dystrophy
19. Update in the Mucopolysaccharidoses
20. Developing standardized corticosteroid treatment for Duchenne muscular dystrophy.
21. Association Study of Exon Variants in the NF-κB and TGFβ Pathways Identifies CD40 as a Modifier of Duchenne Muscular Dystrophy.
22. Increase in Full-Length Dystrophin by Exon Skipping in Duchenne Muscular Dystrophy Patients with Single Exon Duplications: An Open-label Study.
23. Alternate Translational Initiation of Dystrophin: A Novel Therapeutic Approach
24. Exome sequencing in the pediatric neuromuscular clinic leads to more frequent diagnosis of both neuromuscular and neurodevelopmental conditions
25. Phenotypic Spectrum of Dystrophinopathy Due to Duchenne Muscular Dystrophy Exon 2 Duplications
26. Gene editing and modulation for Duchenne muscular dystrophy
27. How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration.
28. Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45.
29. Outcome reliability in non-ambulatory boys/men with Duchenne muscular dystrophy.
30. Variants in MED12L, encoding a subunit of the mediator kinase module, are responsible for intellectual disability associated with transcriptional defect
31. Ataluren treatment of patients with nonsense mutation dystrophinopathy.
32. One year outcome of boys with Duchenne muscular dystrophy using the Bayley-III scales of infant and toddler development.
33. CRISPR/Cas9 homology-independent targeted integration of exons 1-19 restores full-length dystrophin in mice
34. Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
35. The 100-meter timed test: Normative data in healthy males and comparative pilot outcome data for use in Duchenne muscular dystrophy clinical trials
36. Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes
37. X-linked muscular dystrophy in a Labrador Retriever strain: phenotypic and molecular characterisation
38. Promising AAV.U7snRNAs vectors targeting DMPK improve DM1 hallmarks in patient-derived cell lines
39. Update in Duchenne and Becker muscular dystrophy
40. A genome-wide association analysis of loss of ambulation in dystrophinopathy patients suggests multiple candidate modifiers of disease severity
41. Interim results of Transpher A, a multicenter, single-dose clinical trial of UX111 gene therapy for Sanfilippo syndrome type A (mucopolysaccharidosis IIIA)
42. The first exon duplication mouse model of Duchenne muscular dystrophy: A tool for therapeutic development
43. Young Becker Muscular Dystrophy Patients Demonstrate Fibrosis Associated With Abnormal Left Ventricular Ejection Fraction on Cardiac Magnetic Resonance Imaging
44. 204th ENMC International Workshop on Biomarkers in Duchenne Muscular Dystrophy 24–26 January 2014, Naarden, The Netherlands
45. A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy using rAAVrh74.MCK.GALGT2
46. Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy
47. Exome sequencing identifies a DNAJB6 mutation in a family with dominantly-inherited limb-girdle muscular dystrophy
48. Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects with Duchenne muscular dystrophy: Results of a double-blind randomized clinical trial
49. Cryptic MHC class I-binding peptides are revealed by aminoglycoside-induced stop codon read-through into the 3′ UTR
50. Duchenne and Becker Muscular Dystrophies
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