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Feb. 18, 2016

TOPIC: Research

NORD Awards New Research Grants for Rare Disease Research

Posted by Jennifer Huron

 The National Organization for Rare Disorders (NORD), the leading independent, nonprofit organization committed to the identification, treatment, and cure of rare disorders, has awarded six new research grants to fund rare disease research.

“NORD research grants are funded primarily by patients, patient organizations, families and friends of patients who make possible the study of diseases for which no other funding is available,” said Jacqueline Kraska, NORD Research Programs Manager. “Over the years, NORD grants have led to the development of two FDA-approved treatments and numerous journal articles.”

Research grants awarded for 2015 Requests for Proposals include:

For the study of Creutzfeldt-Jakob Disease (CJD), with support funding raised by the Kate Obstgarten Family Foundation, Lundbeck “Raise Your Hand” Campaign, and public donations:

  • Adriano Aguzzi, M.D., Ph.D., Professor and Director, Institute of Neuropathology, University of Zurich, Switzerland, Analysis of anti-PrP Antibodies as a pilot study in Prion Protein mutation carriers.

For the study of Pseudomyxoma peritonei (PMP), with support funding raised by PMP Research Foundation:

  • Wilbur B. Bowne, M.D., F.A.C.S. (Primary Investigator), Hao Cheng, Ph.D., Assistant Professor (Co-investigator), Drexel University, College of Medicine, Philadelphia, PA, Developing a Novel Drug Delivery Platform for Targeting Hyaluronan
  • Kjersti Flatmark, M.D., Ph.D. (Primary Investigator), Radium Hospital, Oslo University Hospital, Norway, Cure4PMP – genomic biomarkers and actionable targets

For the study of Alveolar Capillary Dysplasia (ACD), with support raised from The David Ashwell Foundation, William & George Akers Private Foundation, Alveolar Capillary Dysplasia Association and public donations:

  • Partha Sen, Ph.D. (Primary Investigator), Aaron Hamvas, M.D. (Co-investigator), Northwestern University, Chicago, IL, Characterizing the FOXF1 gene network in lung development

For the study of Homocystinuria due to Cysathionine Beta-Synthase Deficiency, with support raised from public donations:

  • Yair Ankster, M.D. Ph.D. (Primary Investigator), Sheba Medical Center, Ramat Gan, Israel, High throughput measurement of Homocysteine, the marker for Homocystinuria, in the routine newborn screening panel by direct injection

For the study of Lysosomal Storage Diseases, with support raised from public donations:

  • Natalia Gomez-Ospina (Primary Investigator), Stanford University, Stanford, CA, Correction of Mucopolysaccharidosis type 1: Targeting safe harbor loci using genome editing

NORD will be announcing the 2016 Requests for Proposals in short order. Researchers who are interested in applying should visit NORD’s website: https://rarediseases.org/research-grant-program.

Grants are made possible by allies of the rare diseases community that have generously supported research in rare diseases by donating to a NORD research fund.  After funds mature, NORD issues research requests for proposals, which are then independently reviewed by NORD’s Medical Advisory Committee.

Rare diseases present a significant health care concern: nearly 7,000 rare diseases affect 30 million Americans (1 in 10 nationwide)—the majority of whom are children—yet less than 5 percent of rare diseases have an FDA-approved treatment.

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