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The effect of sodium oxybate (Xyrem ®) in a patient with laryngeal dystonia and voice tremor before (A) and after (B) drug intake (S imonyan K and Frucht SJ, Tremor Other Hyperkin Dis, 2013 ) Current treatment of laryngeal dystonia (LD) is limited to the temporary management of voice symptoms with...

POSTDOCTORAL FELLOWSHIP IN DECISION MODELING AND CANCER SURVIVORSHIP The Division of General Pediatrics at Boston Children’s Hospital is seeking a highly motivated and capable decision scientist, epidemiologist or outcomes researcher with a strong interest in cancer survivorship. The fellow will...

The Division of General Pediatrics at Boston Children’s Hospital is seeking a full-time Senior Scientist with a core competence in Quantitative Clinical Pharmacology and Pharmacokinetics and experience in laboratory management. The successful candidate will conduct and supervise assay development...

Dear Colleagues, Research in gene augmentation and genome editing for the treatment of inherited retinal disorders (IRDs) is thriving. It is estimated that IRDs affect about 200,000- 300,000 people in the United States and 4-6 million people worldwide. Most patients with these conditions become...

Gene-Augmentation Therapy Gene therapy typically refers to gene-augmentation therapy. In this approach, a healthy version of the mutated gene is packaged inside an engineered and nonpathogenic form of adeno-associated virus (AAV), with the virus serving as a vector to carry the gene. AAV does not...

Advances in the future may include viral vectors, non-viral vectors, and delivery methods that allow treatment of a greater retinal expanse and an expanded approach to technical challenges related to gene size and disease mechanisms. Genetic therapies for non-retinal ophthalmic diseases are also...

Wet lab and dry lab postdoc positions at Boston Children’sHospital and Harvard Medical SchoolThe Gutierrez-Arcelus Lab at Boston Children’s Hospital and Harvard Medical School(gutierrezarceluslab.com) is seeking wet lab and dry lab postdoctoral research fellowsto work on systems immunology of immune...