Patients, Supporters Worldwide Recognizing Rare Disease Day 2020
An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening ... Read more
Updates
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Advice on Starting Nonprofit Groups for Rare and Other Diseases Focus of NORD Webinar
Starting a 501(c)(3) tax-exempt nonprofit isn’t easy, but the National Organization for Rare Disorders gave a few tips for those looking to begin the ... Read more -
First Patient Dosed in Phase 2 Trial of Alvocidib for Relapsed or Refractory AML, Tolero Says
The first patient has been dosed in a Phase 2 clinical trial examining Tolero Pharmaceuticals‘ investigational CDK9 inhibitor alvocidib as treatment for people with ... Read more -
FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar
The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. ... Read more -
With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus
The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business ... Read more
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Patients, Supporters Worldwide Recognizing Rare Disease Day 2020
An abundance of events are afoot around the world to mark Rare Disease Day ... -
First AML Patient Dosed in Phase 1 Trial of Cellectis’ CAR T-cell Therapy, UCART123
Cellectis has started dosing participants in its Phase 1 trial assessing the safety and ... -
Computers Can Quickly See Patterns in Data to Diagnose Ills Like AML, Study Suggests
Using artificial intelligence to analyze gene expression patterns — which genes are ‘switched on’ ... -
Sellas Begins Screening AML Patients for Phase 3 Trial Testing Cancer Vaccine Galinpepimut-S
Sellas Life Sciences has started screening acute myeloid leukemia (AML) patients for a Phase ... -
FDA Grants Orphan Drug Status to Precigen’s Investigational PRGN-3006 CAR-T Cell Therapy for AML
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Precigen‘s ... -
Flotetuzumab Puts A Third of Hard-to-treat AML Patients in Remission, Phase 1/2 Data Show
Treatment with MacroGenics‘ investigational molecule flotetuzumab completely eliminated traces of cancer cells in a ...
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