Vilcek Foundation Seeking Applicants for Biomedical Research Award

Mar 7, 2018, Posted in Awards, Outside Opportunities, Science

The Vilcek Foundation is currently seeking applicants for the 2019 Vilcek Prizes for Creative Promise in Biomedical Science. We are accepting applications from foreign-born scientists age 38 and under from now through June 11, 2018. Three winners will each receive a $50,000 unrestricted cash prize and will be honored at an awards ceremony in New York City in April 2019.

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Rare Disease Day at the NIH

Mar 2, 2018, Posted in Collaborations, Science, Synodos

By Salvatore La Rosa

On Feb 29, 2008, EURORDIS, a European rare disease organization, sponsored the first Rare Disease Day in Europe. Then the United States joined the first global observance the following year, along with 23 other countries. This year I was invited to participate at the Rare Disease Day at the National Institutes of Health (NIH), an event that aims to bring together “Patients and Researchers – Partners for Life,” as their slogan has always been.

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Accelerating NF Research Thru Technology

Feb 28, 2018, Posted in Latest News, NF Hero, NF2, Science

By Onno Faber

Rare Disease Day is recognized every year on the on the last day of February. It’s a time for the entire rare disease community to come together to raise awareness with the general public and decision-makers about rare diseases and their impact on patients’ lives. Neurofibromatosis affects 1 in 3,000 people overall; NF2 is even more rare (1 in 25,000) and schwannomatosis even more rare (1 in 40,000). 

We asked NF2 patient Onno Faber to tell us about his journey to a diagnosis and how he leveraged his experience as a Silicon Valley technology entrepreneur to accelerate rare disease research & drug development.

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Selumetinib Granted Orphan Drug Designation by the FDA for NF1

Feb 15, 2018, Posted in Latest News, NF1, Science

AstraZeneca and Merck today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for selumetinib, a MEK 1/2 inhibitor, for the treatment of neurofibromatosis type 1 (NF1). Selumetinib was shown to be effective in shrinking tumors because of an early-stage investment of the Children Tumor Foundation. Over 70% of participants in this MEK clinical trial are showing reductions in tumor size from 20-50% of their inoperable plexiform neurofibromas.

Registration for the 2018 NF Conference is now open

Feb 14, 2018, Posted in Latest News, NF Conference, Science

Join us November 2-6, 2018 in Paris, France for the Joint Global Neurofibromoatosis Conference, a global event attracting more than 800 participants worldwide across a wide range of scientific disciplines, from research and clinical backgrounds, all focused on improving outcomes for patients with neurofibromatosis type 1 (NF1), neurofibromatosis type 2 (NF2) and schwannomatosis.

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