Targeting the Mutation, Not the Disease is Bringing Treatment to the Ultra-Rare Community
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Targeting the Mutation, Not the Disease is Bringing Treatment to the Ultra-Rare Community

The U.S. FDA just announced that it has accepted Genentech’s supplemental New Drug Application (sNDA) and has granted Priority Review for Zelboraf (vemurafenib) in patients with Erdheim-Chester disease from the…

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Could this Great New Resource for the CGD Community Help You?
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Could this Great New Resource for the CGD Community Help You?

Chronic granulomatous disease (CGD) is an inherited primary immunodeficiency disease associated with granulomas, which are clumps of immune cells that form at the site of infection or inflammation. As a…

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Amazing Research Uncovered: Guess What These Scientists are Doing for IPF

Most people take breathing for granted — until the ability to breathe is impaired. People with idiopathic pulmonary fibrosis (IPF) know this all too well. Without the ability to readily…

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This Novel Therapy Could Reverse the Biological Causes of DMD
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This Novel Therapy Could Reverse the Biological Causes of DMD

Mitobridge, Inc., a pharmaceutical company that focuses on improving mitochondrial function, just announced the achievement of a vital milestone in the first-in-human clinical trial of PPAR-delta modulator, MA-0211. This study…

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This WM Patient from Across the Pond Has Been Reconnected With His Donor
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This WM Patient from Across the Pond Has Been Reconnected With His Donor

Waldenstrom macroglobulinemia (WM) is a devastating form of non-Hodgkins lymphoma. Specifically, this cancer starts in the B cells of the body (B lymphocytes). Waldenstrom's cancer cells are capable of making…

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