TORONTO, Canada – JDRF and Sanofi Canada are announcing a partnership aimed at amplifying awareness around type 1 diabetes (T1D), a chronic autoimmune condition, and the role screening could play in the early detection of this disease. Nationally, the number of individuals diagnosed with T1D is growing at an estimated...
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SHANGHAI, China — In another milestone since the implementation of CAR-T therapy at Jiahui, the first myeloma patient treated with CAR-T at Jiahui International Hospital was recently discharged two weeks after infusion. The foreign patient was first diagnosed with multiple myeloma in 2008. Despite multiple treatments including chemotherapy, immunotherapy, target...
Amsterdam, The Netherlands and San Antonio, TX, USA – John P. Cogswell, PhD, has been chosen as the recipient of the 2009 Alzheimer Award presented by the Journal of Alzheimer’s Disease in recognition of his outstanding work, “Identification of miRNA changes in Alzheimer’s disease brain and CSF yields putative biomarkers...
Johns Hopkins Medicine (JHM) has been designated a National Organization for Rare Disorders (NORD) Rare Disease Center of Excellence. The newly established designation recognizes centers who are leaders in the diagnosis and care of people with rare diseases. JHM shares the recognition with the Kennedy Krieger Institute. Each year, clinicians...
Moebius Syndrome Foundation organizes this special holiday happy hour for all adults (ages 18+) that have Moebius syndrome. It is a great opportunity to connect with others play some games and get festive! This event is not focused on any one holiday but celebrates all holidays in the month of...
CAMBRIDGE, Mass. & SALISBURY, England – KalVista Pharmaceuticals, Inc. (NASDAQ: KALV), a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of small molecule protease inhibitors, announced today that Nancy Stuart has been appointed to its Board of Directors effective March 18, 2021. Ms. Stuart is Chief Operating...
House Minority Leader Bradley H. Jones, Jr. (R-North Reading) has appointed State Rep. Hannah Kane (R-Shrewsbury) to serve as his designee on the newly-established Rare Disease Advisory Council. During the 2019-2020 legislative session, Kane was the lead co-sponsor, along with Representative Joseph McKenna (R-Webster), of legislation calling for the creation...
NEWTON, Mass. – Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, today announced the appointment of Richard Paulson as Karyopharm’s President and Chief Executive Officer, effective May 3, 2021. Mr. Paulson will also remain a member of the Board of Directors. He will succeed Michael G....
PEA RIDGE – Kyler Kasper is living on borrowed blood. He has not yet celebrated his second birthday, but Kyler’s parents expect him to reach it, thanks to blood donations from strangers. Kyler was recently diagnosed with Diamond Blackfan anemia, a rare blood disorder that affects about 500 people in...
Brussels, Belgium – Patients are central to the rare disease community on all levels. This sentiment was echoed by representatives from all stakeholder groups at the 2023 World Orphan Drugs Congress which took place in early November 2023 in Barcelona. The Parliament joined the Alexion – AstraZeneca Rare Disease team...