European Myasthenia Gravis Day will make it to the calendar for the very first time on 2 June 2023. A symbolic date that will shine the light on this neuromuscular autoimmune disease that affects between 56,000 up to almost 100.000 people in Europe1. This day, initiated by the 'All United for MG'2 coalition which unites patient associations and representatives from seven countries across Europe, aims to raise awareness of the disease and its consequences for patients and their caregivers amongst the general public, medical professionals and the European institutions.
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SEATTLE and SAN RAFAEL, Calif. – The Allen Institute and BioMarin Pharmaceutical Inc. announced a collaboration today that will use technologies developed at the Allen Institute to create new gene therapies aimed at rare genetic diseases of the central nervous system. BioMarin will receive an exclusive license to each program...
Chicago, Illinois – Patients with muscle-invasive urothelial cancer and a high risk of recurrence after surgery may have a new treatment option. The Alliance for Clinical Trials in Oncology today announced positive results from the phase III AMBASSADOR (A031501) trial for the adjuvant treatment of patients with localized muscle-invasive urothelial...
In a recent study, patients with myelodysplastic syndrome (MDS) who have myelofibrosis (MF) showed a worse prognosis when MF was of greater severity. However, this pattern of severity influencing prognosis appeared negated with allogeneic hematopoietic stem cell transplantation (allo-HSCT). The study findings were reported in the journal Experimental Hematology & Oncology....
SAN FRANCISCO – AllStripes, a healthcare technology company dedicated to accelerating treatment research for patients with rare diseases, today announced a collaboration with the National Center for Advancing Translational Sciences (NCATS), an institute of the NIH established to transform the translational process so that new treatments and cures for disease...
PARIS, France and CAMBRIDGE, MA, USA – Sanofi and Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced a strategic restructuring of their RNAi therapeutics alliance to streamline and optimize development and commercialization of certain products for the treatment of rare genetic diseases. Specifically: Sanofi will obtain global development and commercialization rights...
CAMBRIDGE, Mass. – Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that it has started a clinical study of a biannual dosing regimen of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis. The study is being conducted during a randomized treatment extension...
CAMBRIDGE, Mass. – Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today positive early results on clinical outcome measures from the 12-month analysis of ILLUMINATE-A Phase 3 study of OXLUMO® (lumasiran), an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) –...
CAMBRIDGE, Mass. – Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that pivotal trial results from the ILLUMINATE-A Phase 3 study of lumasiran, an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – for the treatment of primary hyperoxaluria type 1 (PH1), were published online in...
CAMBRIDGE, Mass. – Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced that pivotal trial results from the ILLUMINATE-A Phase 3 study of lumasiran, an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – for the treatment of primary hyperoxaluria type 1...