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National Organization for Rare Disorders to Honor Hetlioz

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On May 19, the National Organization for Rare Disorders (NORD) will honor the patients, leaders, and innovators who are moving the rare disease community forward at its annual Portraits of Courage Gala in Washington DC. NORD is a nonprofit organization dedicated to helping patients with rare diseases. (A rare disease is defined as a disease that affects fewer than 200,000 in the US).

Among the honorees are Vanda Pharmaceuticals Inc for its pharmaceutical Hetlioz, which will be honored with an Industry Innovation Award. Hetlioz, which was Food and Drug Administration (FDA) approved in January 2014, treats Non-24-Hour Sleep-Wake Disorder.

The other 2015 honorees include:

Abbey S. Meyers Leadership Award

  • The International Pemphigus & Pemphigoid Foundation

Industry Innovation Award

  • BioMarin Pharmaceutical Inc. – Vimizim (Mucopolysaccharidosis Type IVA, also known as Morquio A Syndrome)
  • BOEHRINGER-INGELHEIM Corporation USA – Ofev (Idiopathic Pulmonary Fibrosis)
  • Genzyme Corporation – Cerdelga (Gaucher Disease Type I)
  • Lundbeck – Northera (Neurogenic Orthostatic Hypotension)
  • Novartis Pharmaceuticals – Zykadia (Anaplastic Lymphoma Kinase-Positive Metastatic Non-Small Cell Lung Cancer)

Lifetime Achievement Award

  • Francis S. Collins, MD, PhD—director, National Institutes of Health

National Health Leadership Award

  • The Honorable Lamar Alexander (TN), US Senate
  • The Honorable Robert Casey, Jr. (PA), US Senate

Portraits of Courage Award

  • Devin Alvarez (Sprengel’s Deformity)
  • Emily Argersinger (Sturge-Weber Syndrome)
  • Laura Crandall (Sudden Unexplained Death in Childhood)
  • Anthony Ferrandino (Batten Disease)
  • Bailey Gribben (Neurofibromatosis)
  • Sophia Hanson (Lymphedema Praecox)
  • Savannah Hollis (Cavernous Angioma)
  • Glenn and Cara O’Neill (Sanfilippo Syndrome Type A)
  • Yusuf Patel (Methyl Melonic Acidemia)
  • Lori Sames (Giant Axonal Neuropathy)

Rare Disease Public Awareness Award

  • Cindy Abbott (Wegener’s Granulomatosis)

Special Recognition

  • Margaret A. Hamburg, MD—former commissioner, Food and Drug Administration

The funds raised will support the organization’s mission of providing education, advocacy, patient support and research for the 30 million Americans with rare diseases. More than 500 guests are expected to attend.

“We look forward to celebrating the community’s accomplishments and the people and organizations that made them possible,” says Peter L. Saltonstall, president and CEO of NORD, in a release. “The rare disease community is exemplified by remarkable courage and determination.”

Current sponsors include Genzyme Corporation, BioMarin Pharmaceutical Inc, Genentech, Aegerion Pharmaceuticals, Lundbeck, Onyx Pharmaceuticals, Shire, Alexion Pharmaceuticals, Alnylam Pharmaceuticals Inc, Baxter International Inc, CSL Bering, GSK (GlaxoSmithKline), Hyperion Therapeutics, Inc, Vanda Pharmaceuticals Inc, Cytokinetics, Inc, Dohmen Life Science Services, Stealth BioTherapeutics Inc. Rare Disease Report is a media partner for the event.