Alnylam Pharmaceuticals announced a potential new biannual dosing regimen for vutrisiran, its investigational treatment for familial amyloid polyneuropathy (FAP) and other forms of ATTR amyloidosis. In its “RNAi Roundtable” webinar, the company also stated that it remains on track to announce top-line data from HELIOS-A…
Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…
Vyndaqel (tafamidis meglumine) remains safe after more than three years of treatment in patients with familial amyloid polyneuropathy (FAP), with no new safety concerns raised, according to an early analysis of a clinical trial in Japan. The study, “Characteristics of Patients with Hereditary Transthyretin Amyloidosis…
Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…
Using ultrasound to measure the size of the median nerve — which runs down the upper arm and continues into the forearm and hand — could be useful for the early diagnosis of familial amyloid polyneuropathy (FAP), a new study indicates. The study, “Nerve ultrasound…
The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis Scholarship Fund will include 32 scholarships — each totaling $5,000 —…
Treatment with Tegsedi (inotersen) eased a range of neuropathy symptoms in people with familial amyloid polyneuropathy (FAP), new data from the NEURO-TTR Phase 2/3 trial show. The findings, “Neuropathy Symptom and Change: Inotersen Treatment of Hereditary Transthyretin Amyloidosis,” were published in the journal Muscle & Nerve,…
When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films to an audience. Co-founders Daniel DeFabio and Bo Bigelow, who are both fathers of children with…
Treatment with Tegsedi (inotersen) slows neurological disease progression in people with familial amyloid polyneuropathy (FAP), results from a Phase 2/3 clinical trial show. The findings may be useful in designing optimal tools for measuring response to treatment in FAP, researchers said. The study, “Mnis+7 And…
Long-term treatment with Onpattro (patisiran) safely and effectively halts or reverses disease progression in people with familial amyloid polyneuropathy (FAP), final data from a two-year Phase 2 trial show. The findings were consistent with results from the Phase 3 APOLLO trial (NCT01960348), which supported Onpattro’s approval…