News

December 5, 2019December 5, 2019

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

News

Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking ... Read more

November 15, 2019November 16, 2019

Rare Disease Film Festival Highlights Patient and Researcher Unity

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Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: ... Read more

November 12, 2019November 12, 2019

Quizartinib Not Recommended in Europe as Therapy for Relapsed or Refractory AML with FLT3-ITD Mutation

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The Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency (EMA), has issued a negative opinion on quizartinib, being developed by Daiichi Sankyo. CHMP ... Read more

November 5, 2019December 20, 2019

Bemcentinib Earns FDA Fast Track Designation for Elderly Patients with Relapsed AML

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The U.S. Food and Drug Administration (FDA) has granted fast track designation to BerGenBio‘s bemcentinib as a treatment for elderly people with acute myeloid leukemia (AML) whose disease has relapsed, ... Read more

October 29, 2019

European Commission Approves Xospata for AML Patients with Certain Genetic Mutation

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The European Commission has approved Astellas Pharma‘s small molecule inhibitor Xospata (gilteritinib) for the treatment of relapsed or refractory acute myeloid leukemia (AML) with a FLT3 mutation, the company has ... Read more

January 3, 2019January 3, 2020

CC-486 as Maintenance Therapy Extends Survival in AML Patients, Phase 3 Trial Shows

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People with acute myeloid leukemia (AML) who are in complete remission after their first-line chemotherapy and not suitable for a stem cell transplant have significantly better survival outcomes if offered ... Read more