Researchers have analysed image and genomic data from the UK Biobank to find insights into rare diseases of the human eye. These include retinal dystrophies – a group of inherited disorders affecting the retina – which are also the leading cause of blindness certification in working-age adults. The retina is...
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BALA CYNWYD, Pa. — Larimar Therapeutics, Inc. (Nasdaq: LRMR), a biotechnology company focused on developing treatments for complex rare diseases, today announced dosing of the first patient in an open label extension (OLE) study evaluating 25 mg daily subcutaneous injections of nomlabofusp in participants with Friedreich’s ataxia (FA). Nomlabofusp (CTI-1601)...
BALA CYNWYD, Pa. — Larimar Therapeutics, Inc. (“Larimar”) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, announced preliminary top-line data from the 25 mg cohort of its Phase 2, four-week, placebo-controlled, dose exploration trial of CTI-1601 in participants with Friedreich’s ataxia (FA). Participants in...
SAN DIEGO – Lassen Therapeutics a biotechnology company developing antibodies as potential treatments for fibrosis and oncology, today announced a collaboration with Cedars Sinai Medical Center. The collaboration will study the role of IL-11 signaling and its inhibition in lung fibrosis, particularly idiopathic pulmonary fibrosis (IPF), and the potential therapeutic effect...
MARLBOROUGH, Mass. – Boston Scientific Corporation (NYSE: BSX) announced positive data from the TARGET study of the TheraSphere™ Y-90 Glass Microspheres (TheraSphere) – a type of radioembolization comprised of millions of microscopic glass beads containing radioactive yttrium (Y-90) – during a late-breaking clinical trial presentation at the annual scientific meeting...
MARLBOROUGH, Mass. – Data presented at hotline and late-breaking trial sessions today at the EuroPCR 2021 congress demonstrated positive procedural performance, including low rates of paravalvular leakage (PVL) and permanent pacemaker implementation (PPI), of the Boston Scientific (NYSE: BSX) ACURATE neo2™ Aortic Valve System within the investigator-initiated Early neo2 Registry...
South San Francisco, Calif. – A new study in BMC Pregnancy and Childbirth finds that objective information about risk of preeclampsia could be key to driving patient behavior change and creates motivation among pregnant patients to follow provider recommendations on prevention, even among those who are medication-hesitant. Key findings...
NORD is excited to be hosting a series of in-person and virtual community listening sessions exclusively for Latino/a/x people affected by rare diseases in the United States. We invite you to help spread the word about this important opportunity by sharing this programming across your network and encouraging participation amongst...
WALTHAM, Mass. — Decision Resources, one of the world’s leading research and advisory firms for pharmaceutical and healthcare issues, finds that the approval and launch of highly anticipated oral agents as well as the increasing use of current and emerging injectable drugs will drive the overall multiple sclerosis drug market...
Utrecht, The Netherlands and Philadelphia — LAVA Therapeutics N.V. (Nasdaq: LVTX), a clinical-stage immuno-oncology company focused on developing its proprietary Gammabody™ platform of bispecific gamma-delta T cell engagers, announced the appointment of Christy J. Oliger to its board of directors effective March 9, 2023. Ms. Oliger brings extensive commercial, portfolio...