A newly developed second-generation transthyretin (TTR) protein stabilizer may be more effective at treating forms of TTR amyloidosis (ATTR), including familial amyloid polyneuropathy (FAP), than one already tested in patients, scientists in Spain announced. Called PITB, the molecule was seen to be better than tolcapone — a TTR…
Intellia Therapeutics is launching a pivotal Phase 3 clinical trial in the U.S. to evaluate the safety and effectiveness of its investigational gene-editing therapy NTLA-2001 in people with ATTR amyloidosis with cardiomyopathy (ATTR-CM). The study, which is expected to start by the end of the year, follows the recent…
The U.S. Food and Drug Administration (FDA) has rejected Alnylam Pharmaceuticals‘ application seeking the expansion of its Onpattro (patisiran) label to include treatment of people with ATTR amyloidosis with cardiomyopathy (ATTR-CM), or heart damage. Based on the regulatory rejection, Alnylam has decided to no longer pursue an approval of…
Attralus’ AT-01 may be more sensitive than a standard imaging agent at detecting toxic transthyretin (TTR) protein clumps in the heart of people with hereditary ATTR amyloidosis, a group of conditions that includes familial amyloid polyneuropathy (FAP), according to new data from a clinical trial. The findings suggest that…
More than a year of treatment with the experimental therapy eplontersen significantly reduces blood levels of transthyretin — the protein that accumulates to toxic levels in familial amyloid polyneuropathy (FAP) — eases nerve damage-related disability, and improves quality of life for patients. That’s according to the full results…
Testing the ability to sense temperatures and mechanical forces could help predict when symptoms will appear for people who carry mutations that cause familial amyloid polyneuropathy (FAP). That’s according to “Quantitative sensory testing and skin biopsy findings in late-onset ATTRv presymptomatic carriers: Relationships with predicted time of disease…
A committee of the U.S. Food and Drug Administration (FDA) has endorsed Onpattro (patisiran) for the treatment of the ATTR amyloidosis with cardiomyopathy (ATTR-CM), or heart damage. Earlier this year, Alnylam Pharmaceuticals, which is developing Onpattro, submitted a supplemental new drug application (sNDA) to the federal agency…
An international panel of patient advocates and healthcare providers has published the first set of recommendations outlining steps for the holistic care of people living with hereditary transthyretin-mediated amyloidosis (hATTR), a group of disorders that includes familial amyloid polyneuropathy (FAP). The guidelines focus not only on early diagnosis…
A rare mutation in the TTR gene was identified as the cause of familial amyloid polyneuropathy (FAP) in a family in China whose first symptom was diarrhea, according to a case report. All the affected family members eventually developed muscle weakness and some had numbness and/or deficient control of…
Treatment with Vyndaqel (tafamidis) reduces signs of heart damage for most people with hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM) associated with the A97S mutation, the most common cause of the disease in Taiwan, a study in that country shows. Notably, improvements in heart health were most pronounced among patients…