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28 results on '"Tawil, Rabi"'

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1. Safety and efficacy of losmapimod in facioscapulohumeral muscular dystrophy (ReDUX4): a randomised, double-blind, placebo-controlled phase 2b trial

2. Engineered FSHD mutations results in D4Z4 heterochromatin disruption and feedforward DUX4 network activation

6. Generation of genetically matched hiPSC lines from two mosaic facioscapulohumeral dystrophy type 1 patients

7. Clinical practice considerations in facioscapulohumeral muscular dystrophy Sydney, Australia, 21 September 2015

8. 225th ENMC international workshop

9. Developing standardized corticosteroid treatment for Duchenne muscular dystrophy

11. Mutations in DNMT3B Modify Epigenetic Repression of the D4Z4 Repeat and the Penetrance of Facioscapulohumeral Dystrophy

15. The FSHD2 Gene SMCHD1 Is a Modifier of Disease Severity in Families Affected by FSHD1

17. Generation of Isogenic D4Z4 Contracted and Noncontracted Immortal Muscle Cell Clones from a Mosaic Patient

18. If you build a rare disease registry, will they enroll and will they use it? Methods and data from the National Registry of Myotonic Dystrophy (DM) and Facioscapulohumeral Muscular Dystrophy (FSHD)

19. DUX4 Activates Germline Genes, Retroelements, and Immune Mediators: Implications for Facioscapulohumeral Dystrophy

23. An expanded version of the Hammersmith Functional Motor Scale for SMA II and III patients

26. Mutations in Kir2.1 Cause the Developmental and Episodic Electrical Phenotypes of Andersen's Syndrome

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