January 21, 2021January 21, 2021 Potentially Less Toxic Chemo for AML Named Orphan Drug in Europe Aspacytarabine (BST-236), a possibly less toxic form of chemotherapy being developed by Biosight for people with acute myeloid leukemia (AML), has been designated an orphan ... Read more
January 21, 2021January 21, 2021 NORD’s Caregiver Respite Program Continues Through Pandemic Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet ... Read more
January 19, 2021January 19, 2021 Art Contest Salutes Creativity of Rare Disease Communities Butterflies, ballerinas, and abstract flowers are featured among the winning artwork of this year’s Rare Artist contest, hosted by the EveryLife Foundation for Rare ... Read more
January 18, 2021January 18, 2021 Iomab-B Shows Promise as Transplant Conditioning Regimen Iomab-B shows promise as a conditioning regimen for bone marrow transplant in patients older than 55 with active relapsed or refractory acute myeloid leukemia ... Read more
January 14, 2021January 14, 2021 Canada Approves Onureg, Oral Chemotherapy for AML Patients in Remission Health Canada has approved Onureg, an oral formulation of the chemotherapy azacitidine, as the first maintenance therapy for adults with acute myeloid leukemia (AML) who have ... Read more
News, syndicated NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum January 11, 2021January 11, 2021 The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life ...
News Ascentage Pharma’s APG-2575 Earns Orphan Drug Designation January 7, 2021January 7, 2021 APG-2575, an investigational Bcl-2 inhibitor being developed by Ascentage Pharma, has received orphan drug ...
News CPI-613 on FDA Fast Track to Treat AML January 4, 2021January 4, 2021 CPI-613 (devimistat) has been granted a fast track designation by the U.S. Food and ...
News NexImmune, City of Hope Partner to Research NEXI-001, New Therapies December 21, 2020December 21, 2020 NexImmune and City of Hope have teamed up on a research initiative aimed at ...
News Venclexta Combos Approved in Canada to Treat Newly Diagnosed AML December 17, 2020December 17, 2020 Health Canada has approved Venclexta (venetoclax) in combination with Vidaza (azacitidine) or low-dose cytarabine to ...
News Potential Cancer Vaccine DCP-001 May Control AML Residual Disease December 14, 2020December 17, 2020 DCP-001, the lead cancer vaccine candidate by DCprime, safely elicited a tumor-specific immune response ...
OSU Names Leukemia Research Center in Honor of Scientist Clara Bloomfield December 10, 2020December 10, 2020 Clara D. Bloomfield dedicated her career to finding new treatments for patients with acute ...
First-in-Human Trial Testing NKX101 Natural Killer Cell Therapy Treats First Patient December 7, 2020December 7, 2020 The first patient has received treatment in a first-in-human Phase 1 trial of NKX101, ...
NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting November 30, 2020November 30, 2020
Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe November 23, 2020November 23, 2020
Dana-Farber Scientist Awarded $400K to Seek New Therapies for Childhood AML November 19, 2020November 19, 2020 Kimberly Stegmaier, MD, of the Dana-Farber Cancer Institute, has been awarded a $400,000 research ...
Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry November 17, 2020November 17, 2020
US Trial Testing Mylotarg Plus Venclexta for AML Now Enrolling Participants November 16, 2020November 16, 2020
1st Patient Dosed in Phase 1/2 Trial Testing CAR T-cell Therapy MB-102 in AML November 9, 2020November 9, 2020 The first patient has been dosed in an open-label Phase 1/2 clinical trial evaluating ...
NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits November 2, 2020November 5, 2020
News Dosing Begins in Phase 1 Trial Testing PRT1419 for Advanced AML, Other Blood Cancers October 29, 2020October 29, 2020 The first patient has been dosed in a Phase 1 trial testing the investigational ...
News, syndicated UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner October 26, 2020October 28, 2020 A photo of a bespectacled young boy, his red baseball cap slightly askew as ...
News FDA Approves Venclexta Combinations for Newly Diagnosed AML October 22, 2020October 22, 2020 The U.S. Food and Drug Administration (FDA) has granted full approval to Venclexta (venetoclax), ...
News, syndicated Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say October 19, 2020October 19, 2020 A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience ...
News Omidubicel Shows Further Trial Benefits for Marrow Transplants in AML, Other Cancers October 15, 2020October 15, 2020 Omidubicel, an experimental cell therapy for blood cancer patients needing a bone marrow transplant, is superior to ...
News TXNIP Protein Deficiency Linked to Poor Prognosis for AML Patients, Study Finds October 12, 2020October 12, 2020 Low expression levels of the protein TXNIP — thioredoxin interacting protein — was associated ...
News Phase 1 Trial Finishes Dosing 1st AML Patient Group With Actimab-A Plus Venclexta October 8, 2020October 8, 2020 The first group of patients with relapsed or refractory acute myeloid leukemia (AML) has ...
News, syndicated Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos October 8, 2020October 8, 2020 Same But Different, a nonprofit U.K. group that uses art for social change, is ...
News ‘Find the Right Fit’ Aims to Educate, Support People With Secondary AML October 1, 2020October 1, 2020 Jazz Pharmaceuticals, with help from the Myelodysplastic Syndrome (MDS) Foundation and the Cancer Support ...