orphan disease

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orphan disease

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.

New England Journal of Medicine has Publishes Emmaus Life Sciences Endari Phase 3 Trial Results

CMT1A is an orphan disease in which patients suffer from progressive muscle atrophy of legs and arms usually appearing during adolescence and progressively evolving.

Pharnext Updates Progress on Trials of PXT3003 to Treat Charcot-Marie-Tooth

- 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.

European Licensing of AOP Orphan's Polycythemia Vera Treatment Ropeginterferon alfa-2b Resumes

- 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.

Synteract Tightens Focus, Updates Brand Platform

"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."

Israel : BIOBLAST PHARMA names Theresa Stevens as new CCDO

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 names 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.

Minoryx Therapeutics names new chief medical officer

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."

United States : SOLIGENIX acquires novel orphan drug candidate SGX301

The company is developing a clinical stage pipeline of orphan disease programmes.

Eiger BioPharmaceuticals concludes merger agreement with Celladon

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.

Amgen partners with Boston Children's Hospital to find new genes and drug targets for severe pain syndromes

MCL is an orphan disease. Orphan diseases are characterized by high unmet need and small patient populations affecting fewer than 200,000 people.

United States : IMBRUVICA (ibrutinib) Capsules Now Approved in the U.S. for Mantle Cell Lymphoma Patients Who Have Received at Least One Prior Therapy

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.