Author Archives: Iqra Mumal, MSc

Review Details Recommendations to Improve FAP Diagnosis

Diagnosing hereditary amyloid transthyretin (ATTRv) amyloidosis with polyneuropathy, also called familial amyloid polyneuropathy (FAP), is challenging, particularly in in places where the disease is less prevalent, researchers say. A high level of suspicion on the part of the physician is required to diagnose patients as early as possible, according…

FAP Annual Costs and Burden Are Considerable, But in Line with Other Rare Diseases

The annual costs and disease burden associated with hereditary transthyretin amyloidosis polyneuropathy (ATTRv-PN), also known as familial amyloid polyneuropathy (FAP), are considerable but within the range of other rare diseases, a Portuguese study shows. The study, titled “Societal costs and burden of hereditary transthyretin amyloidosis polyneuropathy,” was published recently…

Tegsedi Approved in US for Treatment of FAP in Adults

The U.S. Food and Drug Administration has granted approval to Tegsedi (inotersen), by Akcea Therapeutics and Ionis Pharmaceuticals, for the treatment of adults with hereditary transthyretin-mediated amyloidosis (hATTR), also called familial amyloid polyneuropathy (FAP). Along with approval in the U.S., Tegsedi was also approved in Canada this month and in the…

Tegsedi Now Approved in Canada for FAP Treatment in Adults

Tegsedi (inotersen), by Akcea Therapeutics and Ionis Pharmaceuticals, has been granted approval in Canada as a treatment for stage 1 or stage 2 polyneuropathy in adults with hereditary transthyretin (hATTR) amyloidosis, also called familial amyloid polyneuropathy (FAP). This announcement comes at about the same time as Tegsedi’s…

Alnylam Submits New Drug Application in Japan for Patisiran in Treatment of Hereditary ATTR Amyloidosis

Alnylam Pharmaceuticals announced the submission of a New Drug Application (NDA) to Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for  the approval of patisiran as a treatment for hereditary transthyretin-mediated (hATTR) amyloidosis. hATTR amyloidosis, also known as familial amyloid polyneuropathy (FAP), is caused by mutations in the TTR gene. It…