orphan disease


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orphan disease

n.
A disease that is relatively rare, for which the development of drugs is considered to be commercially nonviable.
References in periodicals archive ?
Emmaus Life Sciences is a biopharmaceutical company engaged in the discovery, development and commercialization of innovative treatments and therapies primarily for rare and orphan disease.
CMT1A is an orphan disease in which patients suffer from progressive muscle atrophy of legs and arms usually appearing during adolescence and progressively evolving.
- Vienna, Austria-based orphan disease pharmaceutical specialist company AOP Orphan Pharmaceuticals' submission for marketing authorisation of Ropeginterferon alfa-2b for treatment of polycythemia vera (PV) in the EU has resumed after clock-stop, the company said.
- Carlsbad, California-based contract research organisation (CRO) Synteract has enhanced its focus with the creation of therapeutic centers of development that include some of the most progressive in the biopharma industry: oncology, especially immunotherapy studies, neuro-degenerative disorders, pediatrics, and rare and orphan disease, the company said.
"With our expansive drug product portfolio and pioneering efforts in a number of orphan disease arenas we have significant potential to accelerate and create value for patients and shareholders through our corporate development efforts."
It was reported on Friday that Minoryx Therapeutics, a Spain-based orphan disease specialist, has named Dr Uwe Meya as its new chief medical officer.
Minoryx Therapeutics, a Spanish orphan disease specialist, has named Dr Uwe Meya as its new chief medical officer, it was reported on Friday.
We also anticipate the potential for a number of federal funding opportunities for SGX301 in this orphan disease, as well as for expansion into other indications of unmet medical need."
The company is developing a clinical stage pipeline of orphan disease programmes.
This partnership brings Amgen, a world leader in human genetic target validation, together with Boston Children's Hospital's Division of Pain Medicine, the first and most active pediatric pain programme in the world, and its Manton Center for Orphan Disease Research.
MCL is an orphan disease. Orphan diseases are characterized by high unmet need and small patient populations affecting fewer than 200,000 people.
Further, the analyst is focused on the Phase I chikungunya antibody mRNA-1944 data in Q4 for read-through to Moderna's orphan disease pipeline.