In this issue: Patient and Family Conferences in June and July | Global PNH Patient Registry Alert | Research/FDA Approvals in the News | A Special Father's Day | New Webinars | Support Groups | Aplastic Anemia Clinical Trial | MDS Update
You're invited! Sessions have been designed for patients, family members and caregivers who are affected by bone marrow failure disease including aplastic anemia, PNH, MDS, MPNs, and other bone marrow failure diseases.
Have you been wondering how to get involved with PNH research but did not know where to start? Here’s your chance! Check out the Global PNH Patient Registry at pnh.iamrare.org
NEW FDA APPROVALS!
U.S. Food and Drug Adminstration (FDA) has approved Empaveli (pegcetacoplan) injection to treat adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare, life-threatening blood disease. Empaveli is the first PNH treatment that binds to complement protein C3. Read about it here.
U.S. Food and Drug Administration (FDA) has approved the expanded use of ULTOMIRIS® (ravulizumab-cwvz) to include children (one month of age and older) and adolescents with paroxysmal nocturnal hemoglobinuria (PNH). ULTOMIRIS® , a long-actingC5 inhibitor, is now the first and only FDA-approved medicine for children and adolescents with PNH. Read about it here.
How are you honoring your Father on Sunday? Make a donation today to keep providing Dads with the answers, support, and hope they need.
Make your gift in honor or in memory of your father or someone you admire!
Are you looking for others who are dealing with the same issues that you're experiencing? The Virtual Support Groups are for you!
If you would like to be in a safe place to talk about what you are going through, or if you would like to encourage others, please check out the Virtual Support Groups HERE. Together, we are strong!
A phase I/II Study of REGN7257 (Anti-interleukin 2 receptor subunit gamma [IL2RG] monoclonal antibody) in patients with severe aplastic anemia that is refractory to or relapsed on immunosuppressive therapy. Please click HERE for more information.
AAMDSIF does not recommend, endorse, or make any representation about the efficacy, appropriateness, or suitability of any clinical trial listed on our website. Pharmaceutical company-sponsored content is highlighted only to give additional information about the trial. All trials are listed on https://clinicaltrials.gov/. Always seek the advice of your physician or other qualified health care provider with any questions you may have regarding a clinical trial, and never disregard professional medical advice or delay in seeking it because of something you have read on our website.
Dr. Carraway does a deep dive into the most promising MDS treatments and research presented at the December 2020 ASH meeting including drugs that are in the development pipeline and will soon be available for patients. Watch on-demand here:https://www.aamds.org/webinar/ash-2020-mds-updates
Be sure to register and attend on 6/30/21 at 2pm EDT.
All of our conferences, webinars, educational opportunities, and patient support are made possible from gifts by people like you, friend. Your donation will help us reach more patients and families.
