This post was originally published on this site Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the estimated 7,000 rare diseases known to science have cures or treatments approved by the U.S. Food and Drug Administration (FDA). Raising awareness of those…
This post was originally published on this site The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have already registered to attend the World Orphan Drug Congress (WODC) USA 2019, set to take place at the Gaylord…
This post was originally published on this site About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The March 4-5 meeting, to take place at the Hilton Am Stadtpark Vienna, features 27 speakers on a variety…
This post was originally published on this site A literature review of research performed in specific populations from countries worldwide estimated a heritability of 60 to 70 percent for familial sarcoidosis, a study has found. The study titled “Clinical epidemiology of familial sarcoidosis: A systematic literature review” was published in the journal Respiratory Medicine. While…
This post was originally published on this site Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions of people — estimates run as high as 350 million — they are thought to impact. Across countries, patients, caregivers and advocates will paint faces,…
This post was originally published on this site Monitoring Hodgkin’s lymphoma patients with positron-emission tomography (PET) scans during chemotherapy can help identify those who respond early to a more intense chemotherapy regimen and switch them to a softer, less toxic regimen, helping to avoid adverse side effects while maintaining the same chance of successful treatment,…
This post was originally published on this site The U.S. Food and Drug Administration (FDA) has granted priority review to Revlimid (lenalidomide) in combination with Rituxan (rituximab) for patients with previously treated follicular and marginal zone lymphoma. A decision on whether to approve the treatment combination is expected by June 27. Celgene’s supplemental New Drug Application…
This post was originally published on this site A Phase 1 trial testing the combination of ADCT-402 (loncastuximab tesirine) with Imbruvica (ibrutinib) for advanced diffuse large B-cell lymphoma (DLBCL) or mantle cell lymphoma (MCL) has dosed the first patient. Patient enrollment, for a planned total of 60 participants, is ongoing in the U.S. and Belgium. More information…
This post was originally published on this site The U.S. Food and Drug Administration has granted priority review to Roche‘s polatuzumab vedotin in combination with Rituxan (rituximab) and the chemotherapy bendamustine for the treatment of patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL). The FDA is expected to make a decision on whether to approve…
This post was originally published on this site Health Canada has approved Yescarta (axicabtagene ciloleucel) as a treatment for adults with diffuse large B-cell lymphoma (DLBCL) who have already received two or more lines of systemic therapy. Specifically, the agency’s Notice of Compliance covers relapsed or refractory patients with unspecified DLBCL, primary mediastinal (thymic) large…
