Louis Mushrow suffers from Smith-Magenis syndrome. Sept. 20, 2013— -- Every time Louis Mushrow, 9, hears certain songs, he beings to cry. But his tears aren't related to hearing sad songs, ...
When Janet and Trent Standard welcomed their daughter two years ago, they not only embraced parenthood but soon advocacy after discovering she was born with a rare genetic condition. The Vancouver ...
WASHINGTON, Aug. 3, 2020 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced that the U.S. Food and Drug Administration (FDA) has accepted for priority review Vanda's ...
Pexidartinib is an oral small molecule that is believed to work by inhibiting colony stimulating factor-1 receptor (CSF1R). The applications are supported by positive results from a double-masked, ...
Health Canada Approves Slenyto® - Extended-Release Melatonin Formulation - as a Prescription Drug for Insomnia in Children with Autism Spectrum disorder and Smith Magenis Syndrome ...
Researchers report that restoring RAI1 gene expression levels during an early age-window partially reversed gene expression and social interaction deficits in a mouse model of Smith-Magenis syndrome ...
A publication of the J Speech Lang Hear Res. Purpose: Smith-Magenis syndrome (SMS), a rare, genetically linked complex developmental disorder caused by a deletion or mutation within chromosome 17p11.2 ...
WASHINGTON, May 13, 2020 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced that following the completion of a Type A Meeting with the U.S. Food and Drug Administration ...
To continue reading this content, please enable JavaScript in your browser settings and refresh this page. The decision comes after the D.C. biotech and the FDA ...
Parent hopes to help others by writing a book about a rare genetic disorder Members of a Tualatin family have turned their experience with a son who has a rare genetic syndrome into a self-published ...
To continue reading this content, please enable JavaScript in your browser settings and refresh this page. The company said it’s now evaluating next steps to get ...
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