News

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 a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…

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. The approach, to be refined within living organisms (in vivo studies), aims to deliver a functional copy of…

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, based on a positive recommendation by the EMA’s Committee for Medicinal Products for Human Use,…

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 how their respective organizations came to be, as well as the benefits of creating patient registries and how they can help…

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. A Phase 2 trial testing this antibiotic in SCD patients, in a new formulation for…

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 recommendation by EMA’s Committee for Medicinal Products for Human Use. Orphan designation is given to investigative…

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 living with sickle cell disease (SCD) in Malawi. Malawi is a country located in sub-Saharan Africa. Each year…

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 organizations and research programs to help people with rare diseases.  “The RareLaunch program is…

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 sickle cell disease (SCD), CSL Behring, the medicine’s maker, announced in a press…

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 covers the use of Adakveo in combination with hydroxyurea (also known as hydroxycarbamide) — another approved therapy…