Hello everyone! My name is Claire Bevec and I’m so excited to announce my monthly column that I will be writing for Harmony 4 Hope. I am so thankful for this opportunity to share some of the most beautiful stories about life with a rare disease. Each month I will...
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ZUG, Switzerland — Micreos is pleased to share the positive results of an investigational study: Endolysin inhibits skin colonization by patient-derived Staphylococcus aureus and malignant T cell activation in cutaneous T cell lymphoma, published in the Journal of Infectious Disease, 2023 (https://doi.org/10.1016/j.jid.2023.01.039). The study, led by Prof Niels Ødum, University...
The Cullen Education and Research Fund (CERF) announced the recipients of the first CERF Medical Engineering Prize for ALS Research: Dr. Leigh Hochberg, Dr. Conor Walsh, and Dr. Sabrina Paganoni. Their “BrainGate+SoftRobotics” team from Brown University, Harvard University and Massachusetts General Hospital (MGH) is creating a series of technologies intended...
SAN FRANCISCO & HONG KONG – With more than 100,000 patients losing sight to Bietti’s Crystalline Dystrophy (BCD) worldwide, Invincible Vision and ReflectionBio® have joined forces to preserve vision of those impacted. Today, ahead of World Rare Disease Day on February 28, the patient-led nonprofit and biotech company are launching...
SAN FRANCISCO, Calif. — Invitae (NYSE: NVTA), a leading medical genetics company, today announced a partnership with BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, designed to advance genetics-based drug discovery for rare diseases. The goal of the collaboration is to generate new insights...
GENEVA, Switzerland and AMSTERDAM, The Netherlands — iOnctura, a clinical-stage biotech developing selective cancer therapies against targets that play critical roles in multiple tumor survival pathways, today announces that the US Food and Drug Administration (FDA) has granted permission to proceed with clinical investigations of roginolisib in the United States...
CARLSBAD, Calif. and CAMBRIDGE, Mass. — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) and Biogen Inc. (Nasdaq: BIIB) announced the decision to terminate development of BIIB105 (ION541) an investigational antisense oligonucleotide (ASO) for amyotrophic lateral sclerosis (ALS) based on topline results from the Phase 1/2 ALSpire study. BIIB105 was designed to reduce expression of...
CARLSBAD, Calif. — Ionis Pharmaceuticals, Inc. today announced that it has entered into a license agreement with Otsuka Pharmaceutical Co., Ltd. under which Otsuka obtains exclusive rights in Europe to commercialize donidalorsen, an investigational prophylactic treatment for hereditary angioedema (HAE). Ionis will maintain responsibility for the non-clinical and clinical development...
CARLSBAD, Calif. — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive topline data from the HALOS Phase 1/2a open-label study of ION582 in Angelman syndrome. ION582 was safe and well tolerated in the study and showed encouraging and consistent benefits in individuals living with Angelman syndrome, with the most robust improvements observed...
CARLSBAD, Calif. – Ionis Pharmaceuticals (NASDAQ: IONS) today announced that the company has launched a new grant program that provides funding to U.S.-based researchers whose work advances the understanding of transthyretin amyloidosis (ATTR), facilitates early diagnosis and enhances disease management of patients living with ATTR amyloidosis. The Janice Wiesman Young...