News

November 26, 2020November 25, 2020

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

News, syndicated

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 health and well-being, according to ... Read more

November 24, 2020November 24, 2020

Gates Foundation Funds Studies Into CRISPR/Cas9 Gene Editing for SCD

News

Intellia Therapeutics has received a grant from the Bill & Melinda Gates Foundation to develop sickle cell disease (SCD) treatments using the CRISPR/Cas9 genome editing technology, the company announced. ... Read more

November 19, 2020November 19, 2020

ARU-1801 Gene Therapy Granted Orphan Drug Status in EU

News

The European Medicines Agency (EMA) has granted the designation of orphan drug to ARU-1801, an experimental gene therapy being developed by Aruvant, for the treatment of sickle cell disease (SCD). The decision, ... Read more

November 18, 2020November 18, 2020

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

News, syndicated

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 patient registry. They shared about ... Read more

November 17, 2020November 17, 2020

Rifaximin, Oral Antibiotic, Named Orphan Drug for Potential to Ease Pain Crises

News

The U.S. Food and Drug Administration (FDA) has designated rifaximin, an oral antibiotic, an orphan drug as a potential treatment of pain crises due to sickle cell disease (SCD), according to a press release. ... Read more

November 12, 2020November 12, 2020

FT-4202, Oral SCD Therapy in Clinical Testing, Named Orphan Drug in EU

News

FT-4202, a potential disease-modifying oral treatment for sickle cell disease (SCD) by Forma Therapeutics, has been designated an orphan drug by the European Medicines Agency (EMA). The decision was based on a positive ... Read more

November 10, 2020November 10, 2020

NIH Funds New Project to Improve SCD Care for Children in Malawi

News

A team of researchers led by UNC Project-Malawi has been given a one-year, $500,000 grant to build clinical and research infrastructure that will improve diagnosis and treatment for children ... Read more

November 6, 2020November 4, 2020

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

News, syndicated

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit ... Read more

November 5, 2020November 5, 2020

CSL889 Designated as Orphan Drug in Europe and US for Treating Sickle Cell Disease

News

The European Commission and the U.S. Food and Drug Administration have granted orphan drug status to CSL889, an investigational therapy for the treatment of acute vaso-occlusive crises (VOCs) in ... Read more

November 3, 2020November 3, 2020

EU Approves Adakveo to Treat Pain Crises in SCD Patients, 16 and Older

News

The European Commission has approved Novartis’ Adakveo (crizanlizumab) as a preventive treatment for recurrent vaso-occlusive crises (VOCs) in people, 16 and older, with sickle cell disease (SCD). The approval ... Read more