February 22, 2021August 23, 2022 CRISPR Gene Editing Used to Build 1st Model of AML Progression Blocking early inflammatory and innate immune signaling is a promising therapeutic target in acute myeloid leukemia (AML), as disruptions in these signaling pathways occur early ... Read more
February 18, 2021February 18, 2021 Potential Therapy May Better Prepare Older Patients Before Stem Cell Transplant JSP191, an investigational therapy by Jasper Therapeutics, showed promising efficacy as a conditioning agent to prepare older patients with acute myeloid leukemia (AML) or myelodysplastic ... Read more
February 16, 2021February 17, 2021 Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by ... Read more
February 15, 2021February 15, 2021 FDA Grants Iadademstat Orphan Drug Designation The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Oryzon Genomics‘ iadademstat, an investigational oral therapy for people with acute myeloid ... Read more
February 11, 2021February 11, 2021 Pandemic Won’t Stop Rare Disease Day on Feb. 28 Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness ... Read more
News Eprenetapopt-Azacitidine Combo Shows Promise for AML With TP53 Mutations February 11, 2021February 11, 2021 Eprenetapopt (APR-246), Aprea Therapeutics’ lead cancer therapy candidate, was well-tolerated and showed promising clinical activity ...
News Xospata Conditionally Approved in China for Certain Acute Myeloid Leukemias February 8, 2021February 8, 2021 Xospata (gilteritinib) has been conditionally approved in China to treat adults who have relapsed or ...
News New Combo Therapy Shows Promising Activity Against AML in Lab Studies February 4, 2021February 4, 2021 Combining two potential cancer therapies with distinct modes of action and limited clinical benefits on ...
News, syndicated NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns February 3, 2021February 3, 2021 While progress was made last year on newborn screening and other policy issues critical to ...
News Magrolimab Achieves Positive Results in Ongoing Phase 1b Trial February 1, 2021February 1, 2021 Gilead Sciences’ investigational therapy magrolimab was well-tolerated and effective in untreated acute myeloid leukemia (AML) ...
News FDA Clears Way for Clinical Trial of VOR33 Stem Cell Therapy January 28, 2021January 28, 2021 Vor Biopharma has been cleared in the U.S. to begin clinical trial testing of its ...
COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say January 25, 2021January 25, 2021 The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide ...
Potentially Less Toxic Chemo for AML Named Orphan Drug in Europe January 21, 2021January 21, 2021 Aspacytarabine (BST-236), a possibly less toxic form of chemotherapy being developed by Biosight for people ...
Canada Approves Onureg, Oral Chemotherapy for AML Patients in Remission January 14, 2021January 14, 2021
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 experiences ...
NexImmune, City of Hope Partner to Research NEXI-001, New Therapies December 21, 2020December 21, 2020
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 treat ...
OSU Names Leukemia Research Center in Honor of Scientist Clara Bloomfield December 10, 2020December 10, 2020
News 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, Nkarta‘s ...
News FDA Puts Eprenetapopt on Fast Track for Treating TP53 Mutant AML December 2, 2020December 3, 2020 Eprenetapopt (APR-246), an investigational therapy being developed by Aprea Therapeutics, has been granted fast track designation by ...
News, syndicated NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting November 30, 2020November 30, 2020 To empower and equip members of the rare disease community to engage state leaders in ...
News New $100M Program Aims to Improve Diversity in Clinical Trials November 26, 2020November 30, 2020 A new $100-million program aims to improve the diversity of participants in U.S. clinical trials with ...
News, syndicated Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe November 23, 2020November 23, 2020 The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and ...
News 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 grant ...
News, syndicated Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry November 17, 2020November 17, 2020 Leaders in the U.S. rare disease community came together recently for a webinar to present ...
News US Trial Testing Mylotarg Plus Venclexta for AML Now Enrolling Participants November 16, 2020November 16, 2020 A Phase 1b study testing Mylotarg (gemtuzumab ozogamicin) plus Venclexta (venetoclax) for adults with relapsing or refractory acute ...
News FDA Grants Orphan Drug Designation to Immune-Onc’s IO-202 November 12, 2020November 12, 2020 The U.S. Food and Drug Administration (FDA) has given the designation of orphan drug to ...
