CAMBRIDGE, Mass. – Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis, met all secondary endpoints measured at 18 months in patients with hATTR amyloidosis with...
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CAMBRIDGE, Mass. – Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today positive results from the 6-month primary analysis of the ILLUMINATE-C Phase 3 open-label study of lumasiran, an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – that is being...
CAMBRIDGE, Mass. – Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of the polyneuropathy associated with hereditary transthyretin-mediated (hATTR) amyloidosis, met all secondary endpoints measured at 18 months, including...
CAMBRIDGE, Mass. – Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for vutrisiran, an investigational RNAi therapeutic for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adult patients with polyneuropathy. “hATTR amyloidosis is a rare,...
SUZHOU, China – Alphamab Oncology (stock code: 9966.HK) announced that U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug application (IND) to initiate an open label, multi-center phase II pivotal clinical study (clinical trial No.: KN046-205) in the United States to evaluate the efficacy, safety and tolerability of KN046...
SEATTLE, Wash. — Alpine Immune Sciences, Inc. (NASDAQ: ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for autoimmune and inflammatory diseases, today shared updated clinical data for povetacicept in IgA nephropathy (IgAN) which will be presented as a late breaking poster at the World Congress of Nephrology...
In the midst of a global pandemic, it’s easy to forget that there are other terrible diseases in the world, ones that we know very little about. Consider amyotrophic lateral sclerosis — ALS. Also known as Lou Gehrig’s disease, ALS is an illness whose outcome is never in doubt. The...
WASHINGTON – Following Amylyx’s announcement that it intends to submit a New Drug Application (NDA) for AMX0035, The ALS Association today urges the Food and Drug Administration to approve the treatment for all people with ALS as soon as possible. The Association was an original funder of Amylyx and has...
The brain is indeed a target for treating ALS (amyotrophic lateral sclerosis), Northwestern Medicine scientists have discovered. This flips a long-standing belief that the disease starts in the spinal motor neurons and any therapy would need to target the spine as the key focus. A new Northwestern study shows the degeneration...
Nelson’s eighth annual ALS walk was a huge success, according to organizer Gord Shannon. More than 175 people from across the West Kootenays came out to walk from Lakeside Park to the Nelson airport and back on June 7, which raised around $40,000 for the ALS Society of Canada. “This...