This page shows the latest hATTR news and features for those working in and with pharma, biotech and healthcare.
Both Onpattro and Tegsedi are licensed to treat the ultra-rare disease hereditary transthyretin-mediated amyloidosis (hATTR) in adults with stage 1 or 2 polyneuropathy, or damage to peripheral nerves. ... Gene-silencing is a promising area of medicine
Alnylam's Onpattro is indicated for a different disease ATTR subtype, hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) where patients suffer polyneuropathy complications from the condition. ... Despite the different indications,
While the drugs represent a major step forward in improving the outlook for patients with hATTR, they do represent a major expense for healthcare systems, as they require lifelong treatment. ... Richard A. Jones, SVP Head of Europe for Akcea
Genzyme had previously licensed rights in 2012 to Alnylam’s lead RNAI drug Onpattro (patisiran) for hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis), which became the first drug in the RNAi class ... returning development and
Room for manoeuvre after initial rejection. NICE has issued draft guidance not recommending two new rival treatments for the rare inherited condition hereditary transthyretin-related amyliodsis (hATTR) – Alnylam’s Onpattro and ... Just 150 people in
for tafamidis as a treatment for cardiomyopathy associated with hATTR amyloidosis after reporting positive results in the phase III ATTR-ACT trial. ... Until recently, patients diagnosed with hATTR amyloidosis faced an uncertain future,” says Professor
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Onpattro was approved to treat adults with the. destruction of nerves that results from a rare disease, hereditary transthyretin- mediated (hATTR) amyloidosis, and was launched with a $450, 000 per year ... Standing in its way is a similarly priced
The drug is licensed to treat to adults with stage 1 or 2 polyneuropathy, the destruction of nerves that results from hereditary transthyretin- mediated (hATTR) amyloidosis, a rare, rapidly progressive, fatal ... Neurologists, cardiologists and
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Healthcare is evolving rapidly. To stand out from the crowd requires a potent combination of rich insight, innovative ideas and...