Gastrointestinal (GI) symptoms, particularly unintentional weight loss, occur frequently in people with hereditary transthyretin amyloidosis, also known as familial…
Articles by Forest Ray PhD
The European Commission (EC) has granted orphan drug status to Intellia Therapeutics’ NTLA-2001 gene-editing therapy for transthyretin amyloidosis (ATTR),…
People with hereditary transthyretin (ATTRv) amyloidosis, which includes familial amyloid polyneuropathy, experience multiple medical issues prior to diagnosis, with…
The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected…
The case of a woman of Afro-Caribbean descent with early onset transthyretin (TTR) amyloidosis, who was found to carry a…
The first patient has been dosed in a Phase 1 clinical trial evaluating NTLA-2001, Intellia Therapeutics’ investigational…
Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography…
Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the…
The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure…
The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during…