Madrid, Spain – A few years ago, a patient was cured of multiple myeloma after being treated for hepatitis C, astounding researchers from the group led by Joaquín Martínez, from the H12O-CNIO Haematological Tumours Clinical Research Unit, a collaboration between the Hospital 12 de Octubre (H12O) and the National Cancer...
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The cohort of 7000 rare diseases is complex, heterogeneous and dynamic. Newer conditions are being identified and reported regularly in the medical literature on a day to day basis. For a long time, the medical fraternity, researchers, and policymakers were unaware of the wide spectrum of rare diseases and the...
BETHESDA, Md. – The Cystic Fibrosis Foundation affirmed its support for the PASTEUR Act, a bipartisan proposal that, if passed, will support the development of new antibiotics and promote appropriate use of existing ones. The organization issued the following statement: “New solutions are needed now to bring novel antibiotics into...
Washington, DC – Rare diseases, by their very nature, don’t fit the mold—so neither should the trials for therapies designed to treat them nor the regulatory process to approve them. This was the tone set during a recent panel discussion where Peter Marks, director of the FDA’s Center for Biologics...
MONTREAL, Québec – Neurenati Therapeutics Inc., a biotechnology company dedicated to developing transformative therapies for rare pediatric diseases, is pleased to announce that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to NEU-001, a novel combination therapy leveraging a neurotrophic growth factor for the treatment of Hirschsprung...
Basel, Switzerland – Some pathogens hide inside human cells to enhance their survival. Researchers at the University of Basel, have uncovered a unique tactic certain bacteria use to spread in the body without being detected by the immune system. In their study, they reveal the crucial role of a bacterial...
Results of part 1 of the ESSENCE phase 3 trial, published in The New England Journal of Medicine, demonstrated that a greater proportion of patients with MASH achieved resolution of steatohepatitis with no worsening of liver fibrosis compared to placebo. Patients with MASH also saw statistically significant and superior improvement...
BEERSE, BELGIUM — Janssen-Cilag International NV, a Johnson & Johnson company, today announced that the European Commission (EC) has approved a Type II extension of indication for RYBREVANT®▼ (amivantamab) in combination with chemotherapy (carboplatin and pemetrexed), for the treatment of adults with advanced non-small cell lung cancer (NSCLC) with epidermal...
PARAMUS, N.J. — NS Pharma, Inc., a subsidiary of Nippon Shinyaku Co., Ltd, announced today that the European Commission (EC) has granted orphan drug designation to NS-229, which is being developed for the treatment of the rare disease eosinophilic granulomatosis with polyangiitis (EGPA). The orphan drug designation by the EC...
YONGIN, South Korea — GC Biopharma Corp. announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to its intracerebroventricular (ICV) Enzyme Replacement Therapy (ERT) candidate, GC1130A, designed for Sanfilippo Syndrome type A (mucopolysaccharidosis type IIIA), developed in collaboration with Novel Pharma. GC1130A has previously achieved notable...