BARCELONA, Spain I March 13, 2025 I SpliceBio, a clinical-stage genetic medicines company pioneering Protein Splicing to address diseases caused by mutations ...
LONDON and NEW YORK, March 13, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicines company, today announced financial and ...
Once in custody, the Swiss man admitted to his viral infamy and owned up to his drug use. His alleged partner-in-crime, whom Sascha said he had just met, remains an enigma yet to be identified, ...
The Ministry of Health, Labor, and Welfare (MHLW) in Japan has granted Orphan Drug Designation (ODD) to Lundbeck’s investigational drug, amlenetug, a potential new treatment option targeting Multiple ...
The FDA has granted orphan drug designation (ODD) to rhenium (186Re) obisbemeda for the potential treatment of patients with leptomeningeal metastases (LM) in patients with lung cancer. Rhenium ...
EN001, a novel therapy under development by ENCell, has been granted Orphan Drug Designation by the U.S. FDA for the treatment of Charcot-Marie-Tooth (CMT) disease. Phase 1 clinical trial ...
The global orphan drug market size was USD 154.20 Billion in 2022 and is expected to register a rapid revenue CAGR of 12.3 % during the forecast period. The global orphan drug market is witnessing ...
A disease is classed as rare when it affects fewer than one in 2,000 people. New orphan drugs can provide rare disease patients with hope for a better quality of life when all existing treatment ...
The European orphan drug market is growing and is already comparable in size and scope to the US market. The European Union (EU) counts 36 million rare disease patients, and the European market is ...
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