New pharmacotherapies for genetic neuromuscular disorders: opportunities and challenges-Reference-Cited by-同舟云学术

New pharmacotherapies for genetic neuromuscular disorders: opportunities and challenges

Published:2019-07-02 Issue:8 Volume:12 Page:757-770
ISSN:1751-2433
Container-title:Expert Review of Clinical Pharmacology
language:en
Short-container-title:Expert Review of Clinical Pharmacology

Author:

Ricci Federica¹,Vacchetti Martina¹,Brusa Chiara²,Vercelli Liliana³,Davico Chiara¹,Vitiello Benedetto¹^ORCID,Mongini Tiziana³

Affiliation:

1. Division of Child Neurology and Psychiatry, Department of Pediatrics, Regina Margherita Hospital, and Department of Public Health and Pediatric Sciences, University of Turin, Turin, Italy

2. Dubowitz Neuromuscular Centre, Great Ormond Street Institute of Child Health, University College London, London, UK

3. Department of Neuroscience “Rita Levi Montalcini”; Hospital Città della Salute e della Scienza, University of Turin, Turin, Italy

Publisher

Informa UK Limited

Subject

Pharmacology (medical),General Pharmacology, Toxicology and Pharmaceutics,General Medicine

Link

https://www.tandfonline.com/doi/pdf/10.1080/17512433.2019.1634543

Reference96 articles.

1. Genome Editing of Monogenic Neuromuscular Diseases

2. U.S. 97th Congress. Orphan Drug Act. Public Law 97-414, January 4, 1983.

3. The 2018 version of the gene table of monogenic neuromuscular disorders (nuclear genome)

4. Diagnosis and management of spinal muscular atrophy: Part 1: Recommendations for diagnosis, rehabilitation, orthopedic and nutritional care

5. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management

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