Senator Hatch and Representatives Lance and Butterfield introduced a resolution that heralds the success of the Orphan Drug Act (ODA) and calls for continued support of the legislation.
This year marks the 35th anniversary for the ODA. That means 35 years of increased hope and treatments for the rare disease community. Prior to the ODA, there were only 34 therapies indicated to treat a rare disease. Now, there are over 700 and counting!
A Memorandum of Understanding has been countersigned between RADOIR and Medical Science Faculty of Tehran University for Research & Scientific collaboration
The first Joy “Campaign of RADOIR” with the slogan “Rare but not Alone!” held on 10th Dec, 2018 with the presence of some Comedians, storytellers, children’s favorite show men and clowns.
A university of Houston pharmaceutical scientist is developing a drug which could bring relief to children suffering with Familial Adenomatous Polyposis (FAP), a rare genetic disorder characterized by hundreds if not thousands of colorectal polyps. This hereditary cancer predisposition syndrome occurs in 3 per 100000 live births and, if left untreated, causes colorectal cancer in patients nearly 100 percent of the time. Prior medication for the diseases failed because it increased the risk of heart attacks and death.
Scientists at Scripps Research have found an important immune system-regulating protein in principle could be targeted to treat cancers and chronic viral infections.
The scientists, in a study published November 12 in Nature Chemical Biology, set out to determine the function of a protein, ABHD12, whose absence causes a rare genetic disease featuring a host of brain and nerve problems.