When Nikolas and Ryan Michaud think of their little sister Jillian, they think of the joy she brings to their life. Nikolas says that Jillian's hugs are unmatched, maybe the…
Paul Williamson has always been a fiercely proud father and an advocate for the rare disease community. His son James was born with Smith-Magenis syndrome (SMS), a rare genetic developmental…
Recently, biopharmaceutical company Vanda Pharmaceuticals announced that the FDA granted priority review status for a New Drug Application (NDA) and Supplemental New Drug Application (sNDA) for both liquid and…
Smith-Magenis Syndrome (SMS) is caused by a deletion of the 17p 2,3 chromosome. the condition affects 1 in every 15,000-25,000 individuals. It often results in a sleep disorder, and various mental, physical,…
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