Nashville, Tennessee – Vanderbilt University Medical Center (VUMC) researchers are touting data from a multicenter, international phase 2 clinical trial showing a new, curative treatment for sickle cell disease (SCD). The therapy, nonmyeloablative haploidentical bone marrow transplant (BMT) with thiotepa and posttransplant cyclophosphamide (PTCy), is proving to have equivalent efficacy...
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Amanda Hamacher was in grade school when she began to lose her sight and hearing as a result of a rare genetic disorder called Alstrom syndrome. Now, the 21-year-old cannot see and has severe hearing loss. Yet, she enjoys a newfound sense of freedom thanks to a program sponsored by...
Intestinal permeability and an overgrowth of bacteria in the small intestine are both associated with nonalcoholic fatty liver disease (NAFLD). These findings are revealed in a new study in the June issue of Hepatology, a journal published by John Wiley & Sons on behalf of the American Association for the...
AUSTIN, TX – IntraBio Inc., a leader in the discovery and development of innovative drugs for rare neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved AQNEURSA (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients...
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...
Daix (France) and New York, NY – Inventiva (Euronext Paris and Nasdaq: IVA) (“Inventiva” or the “Company”), a clinical-stage biopharmaceutical company focused on the development of oral therapies for the treatment of metabolic dysfunction-associated steatohepatitis (MASH), today announces the publication in Journal of Hepatology Reports, a peer-reviewed, scientific journal, of results...
COPENHAGEN, Denmark -Genmab A/S(Nasdaq:GMAB) announced today updated data from cohort B1 of the Phase 1/2 RAINFOL-01 study of rinatabart sesutecan (Rina-S®), an investigational folate receptor-alpha (FRα)-targeted, TOPO1 antibody-drug conjugate (ADC) that showed Rina-S 120 mg/m2 every 3 weeks (Q3W) resulted in a confirmed objective response rate (ORR) of 55.6% (95% CI: 30.8-78.5)...
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...