November 30, 2020November 30, 2020 NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the ... Read more
November 26, 2020November 30, 2020 New $100M Program Aims to Improve Diversity in Clinical Trials A new $100-million program aims to improve the diversity of participants in U.S. clinical trials with the ultimate goal of achieving better health outcomes and ... Read more
November 23, 2020November 23, 2020 Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their ... Read more
November 19, 2020November 19, 2020 Dana-Farber Scientist Awarded $400K to Seek New Therapies for Childhood AML Kimberly Stegmaier, MD, of the Dana-Farber Cancer Institute, has been awarded a $400,000 research grant to continue her work to find new treatments for ... Read more
November 17, 2020November 17, 2020 Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and ... Read more
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 ...
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 ...
News 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 ...
News Complete Remission Reported in Patient in Ongoing Trial of APVO436 November 5, 2020November 5, 2020 Aptevo Therapeutics’ investigational therapy APVO436 resulted in a patient’s complete disease remission in an ongoing ...
News, syndicated NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits November 2, 2020November 5, 2020 The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days ...
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 ...
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 ...
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), ...
Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say October 19, 2020October 19, 2020
Omidubicel Shows Further Trial Benefits for Marrow Transplants in AML, Other Cancers October 15, 2020October 15, 2020
TXNIP Protein Deficiency Linked to Poor Prognosis for AML Patients, Study Finds October 12, 2020October 12, 2020
Phase 1 Trial Finishes Dosing 1st AML Patient Group With Actimab-A Plus Venclexta October 8, 2020October 8, 2020
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 ...
‘Find the Right Fit’ Aims to Educate, Support People With Secondary AML October 1, 2020October 1, 2020
First Clinical Trial Launched for AML, Other Blood Cancer Patients With COVID-19 September 28, 2020September 28, 2020
Dosing Begins in Phase 1 Trial Testing Immune-Onc’s IO-202 for Advanced AML, CMML September 24, 2020September 24, 2020
Rigosertib Combo Fails to Prolong Survival in Higher-risk Myelodysplastic Syndromes September 21, 2020September 21, 2020 When given alongside best supportive care, rigosertib failed to prolong the survival of patients ...
FDA Grants Rare Pediatric Disease Status to AML Therapy OXi-4503 September 17, 2020September 17, 2020
French Authorities Advance Phase 3 Trial Testing Sellas’ GPS Cancer Vaccine September 14, 2020September 14, 2020
News, syndicated LLS Highlights Its Support Programs During Blood Cancer Awareness Month September 10, 2020September 9, 2020 Throughout September for Blood Cancer Awareness Month, the Leukemia & Lymphoma Society (LLS) is asking ...
News FDA Approves Oral Chemotherapy Onureg for Newly Diagnosed AML Patients in Remission September 3, 2020September 3, 2020 The U.S. Food and Drug Administration has approved Onureg, an oral formulation of the ...
News, syndicated Same But Different Photo Contest Celebrates People With Rare Diseases August 31, 2020August 31, 2020 Same But Different, a U.K. nonprofit that uses the arts to bring communities together, ...
News Idhifa Combo Fails to Prolong Survival in Older Patients in Phase 3 Trial August 31, 2020August 31, 2020 Idhifa (enasidenib), given alongside best supportive care, failed to prolong overall survival of older ...
News Venclexta Plus Vidaza Increases AML Survival, Study Shows August 27, 2020August 27, 2020 Adding Venclexta (venetoclax) to treatment with Vidaza (azacitidine) significantly prolongs survival in people with ...
News, syndicated Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input August 24, 2020August 24, 2020 Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, ...
News Phase 1 Trial for Potential Oral Therapy CB-5339 for Myeloid Cancers Doses First Patient August 20, 2020August 20, 2020 A Phase 1 clinical trial evaluating Cleave Therapeutics’ experimental oral therapy CB-5339 as a treatment for ...
News, syndicated EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US August 17, 2020August 17, 2020 The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. ...
News, syndicated New Streaming Channel Showcases Rare Disease Films August 10, 2020August 11, 2020 When the COVID-19 pandemic forced the postponement of a rare disease film festival originally ...
