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March 26, 2020March 26, 2020

Actimab-A and Chemo Combo Showing Promise in Advanced AML Patients

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An early trial testing the investigational radioimmunotherapy Actimab-A plus chemotherapy in people with relapsing or refractory acute myeloid leukemia (AML) has begun dosing a final group of patients, Actinium Pharmaceuticals announced. ... Read more

March 26, 2020March 26, 2020

NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum

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The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. ... Read more

March 20, 2020March 20, 2020

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

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Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see ... Read more

March 13, 2020March 23, 2020

Venclexta-Cytarabine Fails to Significantly Prolong Life in Untreated AML Patients, Trial Shows

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First-line treatment with a combination of Venclexta (venetoclax) and a low dose of cytarabine failed to significantly extend the survival of people with acute myeloid leukemia (AML) who were ... Read more

March 10, 2020March 10, 2020

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

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The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still ... Read more

March 6, 2020March 23, 2020

Phase 2 Trial of Annamycin, as Less Toxic Chemo, in Advanced AML Planned

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Moleculin has announced plans to launch a global Phase 2 clinical trial evaluating its candidate liposomal Annamycin as a less toxic and second-line therapy for relapsed or refractory acute myeloid leukemia (AML). If ... Read more

March 5, 2020March 5, 2020

Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare

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In recognition of Rare Disease Day Feb. 29, BioNews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the ... Read more

March 2, 2020March 2, 2020

Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29

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Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, ... Read more

February 28, 2020March 5, 2020

Medigene’s AML Cancer Vaccine Shows Positive Results in Clinical Trial

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A completed Phase 1/2 clinical trial has shown encouraging results for Medigene‘s immune cell vaccine — which uses dendritic cells to “educate” T-cells about their targets — in people ... Read more

February 27, 2020February 27, 2020

Advice on Starting Nonprofit Groups for Rare and Other Diseases Focus of NORD Webinar

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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 complex process in its Feb. ... Read more

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