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…
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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…
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Alnylam Set to Seek Regulatory Approval for Patisiran With Robust Findings in Phase 3 FAP Trial
Alnylam Pharmaceuticals, together with its partner Sanofi Genzyme, has now released the full results from the Phase 3 trial of patisiran — a…
Children’s National Health System no longer treats just kids. Its Rare Disease Institute, launched in April 2017, has partnered with the National Organization for…
Patients with familial amyloid polyneuropathy (FAP) appear to have intact reproductive capacity, even when they have undergone a liver transplant or receive treatment with Vyndaqel…
Familial amyloid polyneuropathy (FAP) is a progressive disorder that can severely impact the quality of life of patients, causing emotional and psychological distress. According…
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,…
More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars…
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)…
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#NORDsummit – Despite Criticism, Orphan Drug Act Is Working to Advance Needed Treatments, FDA Says
As Congress begins debate this week to overhaul the U.S. tax code, lawmakers should leave the Orphan Drug Act (ODA) — and the tax incentives…
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