Forest Ray PhD,  —

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.

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Weight Loss, Other GI Symptoms, Common in FAP, Italian Study Finds

Gastrointestinal (GI) symptoms, particularly unintentional weight loss, occur frequently in people with hereditary transthyretin amyloidosis, also known as familial amyloid polyneuropathy (FAP), according to data from an Italian study. Indeed, more than 80% of the patients in this study reported experiencing at least one GI symptom, a percentage well…

European Commission Grants NTLA-2001 Gene-editing Therapy Orphan Drug Status

The European Commission (EC) has granted orphan drug status to Intellia Therapeutics’ NTLA-2001 gene-editing therapy for transthyretin amyloidosis (ATTR), a group of disorders that also encompasses familial amyloid polyneuropathy (FAP). Orphan drug status comes with benefits designed to ease a potential medicine’s path to market. “This news is…

Patient Records Show ATTRv Amyloidosis Symptoms Years Before Diagnosis

People with hereditary transthyretin (ATTRv) amyloidosis, which includes familial amyloid polyneuropathy, experience multiple medical issues prior to diagnosis, with potential ATTRv markers commonly appearing in the year before, according to a recent study. A better understanding of patient experiences may help speed its recognition and diagnosis. The study, “…