Articles

Dipanjan Basu, PhD, is testing non-melanoma drugs, like the investigational therapy omipalisib (GSK2126458) in cultured patient cells.

Dipanjan Basu, PhD, Highlights Potential of Omipalisib for Neurocutaneous Melanocytosis

Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.
 

Top 5 Rare Disease News of the Week&mdash;October 14, 2018

In a recent webinar, geneticist Debra Regier, MD, provides an overview of the importance of genetic testing in the rare disease space.

Genetic Testing for Rare Diseases with Geneticist, Debra Regier, MD

e FDA grants Fast Track designation to 177Lu-lilotomab satetraxetan (Betalutin) for the treatment of patients with relapsed/refractory follicular lymphoma after at least 2 prior systemic therapies.

FDA Grants Fast Track Designation to Betalutin for Follicular Lymphoma

Congress has voted to pass the Right to Try bill which will provide terminally ill patients with the ability to seek out experimental therapies not yet approved by the FDA.

"Right to Try" Bill Heads to President Trump for Approval

NORD: The Voice of the Community - May 2018

On May 17th, the National Organization of Rare Disorders (NORD) will host its 2018 Rare Impact Awards in Washington, DC at the Andrew W. Mellon Auditorium.

NORD to Celebrate 2018 Rare Impact Awards and 35th Anniversary

Paul Melmeyer, Director of Federal Policy for the National Organization for Rare Disorders (NORD) wants to do his part to ensure that the proper choices are being made on behalf of people affected by rare diseases.

NORD Director of Federal Policy Emphasizes Patient Engagement

In observance of World Rare Disease Day, the U.S. FDA has announced a collaboration with NORD, in which a pilot project would assist medical reviewers in better understanding rare disease patient experiences.

FDA Observes Rare Disease Day with Help from NORD, NIH

NORD's Voice of the Community - February 2018

NORD's Voice of the Community

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