Karyopharm Therapeutics, a pharmaceutical company focusing on novel cancer therapies, has issued a statement via PR Newswire that the FDA has granted regulatory designations to eltanexor, Karyopharm’s Selective Inhibitor…
In an August 3rd, 2022 news release from Syros Pharmaceuticals (“Syros”), the company shared that its product tamibarotene had received a positive opinion for its Orphan Drug designation application.…
Researchers acknowledge that the primary cause of failure of stem cell transplants is disease relapse. This holds true specifically for allogeneic hematopoietic stem cell transplants where a cancer patient…
Webinar: Living With Chronic Fatigue: I'm Not Just Tired July 23, 2022 with Brandi Reeves, MD; Lineberger Comprehensive Cancer Center, UNC-Chapel Hill School of Medicine, Chapel Hill, NC Attend this…
Webinar: Becoming a Partner in Your Care: MDS Support Group June 25, 2022 with Sara M. Tinsley, PhD, APRN, AOCN, Moffitt Cancer Center, Tampa, FL Attend this webinar dedicated to…
The Aplastic Anemia, MDS, and PNH Virtual Support Group June 25, 2022 AAMDSIF is pleased to announce that we will host a support group for patients diagnosed with aplastic anemia,…
Webinar: The MDS Widow: Sharing and Moving Forward June 23, 2022 Rochelle Ostroff-Weinberg, widow of Robert Weinberg, invites you to join her and other MDS widows on International Widows’ Day. To…
The Aplastic Anemia and MDS International Foundation's Transplant for MDS Virtual Support Group June 15, 2022 AAMDSIF is pleased to announce that we will host a support group for patients…
The Aplastic Anemia and MDS International Foundation's Pediatric Parents Virtual Support Group June 14, 2022 This virtual support group is designed to support pediatric parents whose children have been diagnosed…
The Aplastic Anemia and MDS International Foundation's MDS Virtual Support Group June 11, 2022 AAMDSIF is pleased to announce that we will host a support group for patients diagnosed with…
The Aplastic Anemia, MDS, and PNH Virtual Support Group May 28, 2022 AAMDSIF is pleased to announce that we will host a support group for patients diagnosed with aplastic anemia,…
Webinar: Precision Medicine in MDS and AML May 21, 2022 with Rory Shallis, MD, Yale School of Medicine, New Haven, CT Attend this webinar to learn about the role of…
Webinar: Advances in our Understanding of the Management of MDS March 26, 2022 with Michael Keng, MD, University of Virginia, Emily Couric Clinical Cancer Center, Charlottesville, VA Attend this webinar…
Webinar: Optimizing Transfusion Support in MDS February 12, 2022 with Lindsay R. Wilde, MD, Sidney Kimmel Cancer Center and Thomas Jefferson University Hospital, Philadelphia, PA Attend this webinar to learn…
Orphan drug designation is granted to drugs or biologics intending to treat, diagnose, or prevent rare diseases or conditions. Within the United States, a rare condition is one affecting under…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
Karyopharm Therapeutics has just announced that Eltanexor, their investigative therapy for myelodysplastic syndromes (MDS), has been given Orphan Drug Designation by the FDA. This designation means that the FDA believes…
Each year, approximately 1 in every 1 million infants is diagnosed with pediatric myelodysplastic syndromes (MDS), a group of rare disorders characterized by the abnormal or deficient formation of blood…
Webinar: Focus on Understanding Clinical Trials and How to Identify the Right One for You January 22, 2022 with Andrew M. Brunner, MD, Massachusetts General Hospital, Assistant Professor of Medicine,…
The FDA grants the Orphan Drug designation to treatments indicated for rare diseases, which are defined as affecting less than 200,000 people in the U.S. This designation is meant to…
Over the past few weeks, Centenary College has partnered with Be The Match to host a stem cell donor drive. The first two drives were on November 18th and 19th,…
CAR T Cell Therapy in MDS/AML October 16, 2021 Speaker: David A. Sallman, MD, Moffitt Cancer Center This informational webinar will focus on a treatment approach called CAR T-cell therapy,…
In mid-August 2021, biotechnology and pharmaceutical development company Biosight Ltd. shared that it had begun a Phase 2 clinical trial regarding one of its proprietary treatments - and that the…
In the Phase 3 PANTHER clinical trial, also known as the Pevonedistat-3001 study, researchers were evaluating pevonedistat in conjunction with azacitidine for patients with acute myeloid leukemia (AML), myelodysplastic syndromes…
Science Magazine recently joined other news sources in reporting that Bluebird Bio’s Phase 3 clinical trial investigating a treatment for a neurological disease was put on hold by…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
