Updates

August 10, 2020August 11, 2020
New Streaming Channel Showcases Rare Disease Films

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a ... Read more
August 6, 2020August 6, 2020
Biosight’s Less Toxic Chemo BST-236 Wins FDA Fast Track Status for AML

Biosight‘s BST-236 (aspacytarabine), a less toxic form of chemotherapy for acute myeloid leukemia (AML), has received fast track designation from the U.S. Food and Drug ... Read more
August 3, 2020August 3, 2020
Rare Disease Communities Invited to Submit Works to 2020 Rare Artist Contest

To raise awareness of the rare disease communities and celebrate the creativity of its members, the EveryLife Foundation for Rare Diseases is seeking submissions ... Read more
July 30, 2020July 30, 2020
Trial of CPI-613 in Older AML Patients Moves Closer to 1st Interim Analysis

The Phase 3 trial testing Rafael Pharmaceuticals‘ CPI-613 (devimistat) as a treatment for relapsed or refractory acute myeloid leukemia (AML) has enrolled more than ... Read more
July 23, 2020July 23, 2020
Blood Cancer Groups Unite to Award $6.75M to ‘Propel Discoveries’

A program created by three advocacy groups has awarded more than $6.75 million in multiyear grants to nine scientists whose work might drive a ... Read more

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Updates

New Streaming Channel Showcases Rare Disease Films

Biosight’s Less Toxic Chemo BST-236 Wins FDA Fast Track Status for AML

Rare Disease Communities Invited to Submit Works to 2020 Rare Artist Contest

Trial of CPI-613 in Older AML Patients Moves Closer to 1st Interim Analysis

Blood Cancer Groups Unite to Award $6.75M to ‘Propel Discoveries’

New Molecule Shows Promise as Potential Cancer Vaccine for AML

First Patient Dosed in Phase 1 Trial Testing Small Molecule Treatment CA-4948 for AML

Helsinn, Mei Pharma Stop Phase 3 Trial of Pracinostat-Vidaza Combo for AML

FDA Allows Human Trials of Aptose’s CG-806 Oral Compound for AML

Venclexta Combo Continues to Help Untreated AML Patients in Phase 3 Trial

Daurismo Combo Approved in Europe for Select Adults With AML

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FDA’s New ‘Project Patient Voice’ to Share Symptoms Data From Cancer Trials

Cost of Cancer Treatment in US Could Rise by 34% by 2030

Genetic Testing Often Worth the Wait for More Tailored Treatments, Study Says

FDA Approves Mylotarg as First-Line AML Treatment for Children

Moleculin Seeks Two More Test Sites in European Annamycin Trial

Protocol First Wins Funding for Beat AML Clinical Trial

Coalition Will Address Racial Disparities in Rare Disease Communities

Intellia Looking to Bring Its Potential T-cell Therapy for AML into Trials

MacroGenics Plans Registration Study of Flotetuzumab for Hard-to-treat AML Patients

FDA OKs Phase 1 Trial of Antibody, IO-202, to Treat Advanced AML and CMML

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

Venclexta Plus Vidaza Improves Survival for AML Patients, AbbVie Reports

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Annamycin Trial in Poland Permitted to Accelerate Dose Escalation in AML Patients

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Phase 3 Trial Stops Testing Idasanutlin in AML Patients Undergoing Chemo

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

FDA Reviewing CC-486 as Maintenance Treatment of Adult Patients in Remission

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

GT Biopharma Completes Dosing of First Patient in Phase 1/2 Trial of GTB-3550

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

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New Streaming Channel Showcases Rare Disease Films

Biosight’s Less Toxic Chemo BST-236 Wins FDA Fast Track Status for AML

Rare Disease Communities Invited to Submit Works to 2020 Rare Artist Contest

Trial of CPI-613 in Older AML Patients Moves Closer to 1st Interim Analysis

Blood Cancer Groups Unite to Award $6.75M to ‘Propel Discoveries’

New Molecule Shows Promise as Potential Cancer Vaccine for AML

First Patient Dosed in Phase 1 Trial Testing Small Molecule Treatment CA-4948 for AML

Helsinn, Mei Pharma Stop Phase 3 Trial of Pracinostat-Vidaza Combo for AML

FDA Allows Human Trials of Aptose’s CG-806 Oral Compound for AML

Venclexta Combo Continues to Help Untreated AML Patients in Phase 3 Trial

Daurismo Combo Approved in Europe for Select Adults With AML

Subscribe to our mailing list

FDA’s New ‘Project Patient Voice’ to Share Symptoms Data From Cancer Trials

Cost of Cancer Treatment in US Could Rise by 34% by 2030

Genetic Testing Often Worth the Wait for More Tailored Treatments, Study Says

FDA Approves Mylotarg as First-Line AML Treatment for Children

Moleculin Seeks Two More Test Sites in European Annamycin Trial

Protocol First Wins Funding for Beat AML Clinical Trial

Coalition Will Address Racial Disparities in Rare Disease Communities

Intellia Looking to Bring Its Potential T-cell Therapy for AML into Trials

MacroGenics Plans Registration Study of Flotetuzumab for Hard-to-treat AML Patients

FDA OKs Phase 1 Trial of Antibody, IO-202, to Treat Advanced AML and CMML

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

Venclexta Plus Vidaza Improves Survival for AML Patients, AbbVie Reports

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Annamycin Trial in Poland Permitted to Accelerate Dose Escalation in AML Patients

Video Games Connect Chronically Ill Children Isolated at Home, Hospital

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

Phase 3 Trial Stops Testing Idasanutlin in AML Patients Undergoing Chemo

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

FDA Reviewing CC-486 as Maintenance Treatment of Adult Patients in Remission

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

GT Biopharma Completes Dosing of First Patient in Phase 1/2 Trial of GTB-3550

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

Pin It on Pinterest