The Rare Barometer Program Helps Politicians Stay Informed on Issues Important to Rare Disease Patients

Eurordis-Rare Diseases Europe Eurordis-Rare Diseases Europe is an alliance of patient organizations which works to give rare patients a voice. It also strives to spread awareness of rare diseases to…

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A Plasma Shortage is Affecting Common Variable Immune Deficiency Patient’s Access to Treatment

Heather White Heather White is a 41-year-old woman who was diagnosed with common variable immune deficiency (CVID) back in 2004 after fighting frequent bouts of pneumonia. Doctors believe that she…

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Gaucher Disease Patient and Activist Successfully Improves the Care for Other Rare Disease Patients in Her Country

This is the story of a brave journalist who was determined to improve care for rare disease patients in her country, North Macedonia. North Macedonia is a developing country located…

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University of Michigan Professor Creates Website to Provide Scleroderma Patients Resources and Support

Dinesh Khanna, a professor at the University of Michigan has worked heavily with the rare disease scleroderma. Michigan has its own Scleroderma Program, of which Khanna is the director. Through…

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Phase 3 Trial of Experimental Progressive Familial Intrahepatic Cholestasis Drug for Pediatric Patients Begins
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Phase 3 Trial of Experimental Progressive Familial Intrahepatic Cholestasis Drug for Pediatric Patients Begins

According to a story from drugs.com, the biopharmaceutical company Mirum Pharmaceuticals recently announced that they have begun dosing the first patient in a phase 3 clinical trial. This clinical trial…

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New Research Suggests “Nonsense” RNA Segment Might Play Important Role in Ankylosing Spondylitis
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New Research Suggests “Nonsense” RNA Segment Might Play Important Role in Ankylosing Spondylitis

According to a publication from Ankylosing Spondylitis News, a recent study published in BMC Musculoskeletal Disorders suggests that LINC00311, a lengthy RNA molecule, is over-expressed in individuals with ankylosing spondylitis.…

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Researchers Ask Hemophilia Patients to Evaluate Their Quality of Life on Study Questionnaire
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Researchers Ask Hemophilia Patients to Evaluate Their Quality of Life on Study Questionnaire

According to a publication from Hemophilia News Today, an international team of researchers with members from France and the United Kingdom recently conducted a study of over 100 hemophilia patients…

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FDA Amends Original Approval of Ovarian Cancer Drug Zejula, Expanding Availability

According to a publication from OncLive, the Food and Drug Administration (FDA) recently granted priority review status to a supplemental biologics license application for Zejula. Zejula (generic name niraparib) is…

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Multiple Myeloma Drug ‘Xpovio’ Passes With FDA Accelerated Approval
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Multiple Myeloma Drug ‘Xpovio’ Passes With FDA Accelerated Approval

As reported in Biospace, xpovio (selinexor), a drug used to treat relapsed refractory multiple myeloma, received accelerated approval by the Food and Drug Administration this July. About Multiple Myeloma Multiple…

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