Alice Melao,  —

Pfizer’s Vyndaqel (tafamidis) can significantly delay the progression of familial amyloid polyneuropathy, regardless of its severity before treatment or which mutation causes it, a study reports. The research, based on the combined results of two clinical trials, appeared in the European Journal of Neurology. The title of the article is “…

Alnylam Pharmaceuticals’ lead investigational therapeutic patisiran, for the treatment of hereditary ATTR (hATTR) amyloidosis, also known as familial amyloid polyneuropathy (FAP), was granted accelerated assessment by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP). “We are pleased the CHMP has granted accelerated assessment for patisiran…

Ionis Pharmaceuticals has asked the European Medicines Agency (EMA) to approve its lead drug candidate inotersen to treat hereditary TTR amyloidosis (ATTR), also known as familial amyloid polyneuropathy (FAP). The request, known as a Marketing Authorization Application (MAA), is supported by positive data from the Phase 3 NEURO-TRR trial, and…

Familial amyloid polyneuropathy (FAP) is a progressive disorder that can severely impact the quality of life of patients, causing emotional and psychological distress. According to a study that appeared in the Journal of Community Genetics, FAP patients and members of their family could benefit from additional psychological and psychiatric…

Extra copies of DNA in energy-producing cell components known as mitochondria play a role in the development of the Val30Met-mutation form of familial amyloid polyneuropathy, a Portuguese study suggests. Mutations of the TTR gene cause transthyretin-related familial amyloid polyneuropathy, or TTR-FAP, by producing faulty versions of the transthyretin protein. More…

The latest results of the Phase 3 NEURO-TTR study continue to demonstrate the therapeutic potential of inotersen for patients with hereditary TTR amyloidosis (hATTR) with polyneuropathy, also known as familial amyloid polyneuropathy (FAP). The investigational drug developed by Ionis Pharmaceuticals showed clinically meaningful benefits and improved quality of…

Patients with familial amyloid polyneuropathy (FAP) are in a constant pro-inflammatory state, with increased levels of interleukin-6 (IL-6) driven by a mutated form of the protein transthyretin (TTR), which characterizes this disease. These findings were featured in a study published in the journal Scientific Reports. FAP is caused…

The investigative drug inotersen was shown to significantly reduce neurological progression and improve quality of life in patients with familial amyloid polyneuropathy (FAP) – also referred to as hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN). These findings resulted from the Phase 3 NEURO-TTR study (NCT01737398), and were subject of a…