Forest Ray received his PhD in systems biology from Columbia University, where he developed tools to match drug side effects to other diseases. He has since worked as a journalist and science writer, covering topics from rare diseases to the intersection between environmental science and social justice. He currently lives in Long Beach, California.
Novartis and the Bill and Melinda Gates Foundation are collaborating to develop a single-shot gene therapy that would cure sickle cell disease (SCD) without the need to engineer cells outside ... Read more
The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to ... Read more
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 ... Read more
The Bill & Melinda Gates Foundation has awarded a $3.3 million grant to GreenLight Biosciences to develop gene therapies revolving around messenger RNA (mRNA) — the intermediate molecule between DNA ... Read more
Genetic analyses of African patients identified several genes and biological pathways that are associated with disease severity and longevity, and that might serve as new therapeutic targets in sickle cell ... Read more
Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A ... Read more
Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input ... Read more