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Rare Diseases

U.S. FDA approves drug to treat seizures associated with Lennox-Gastaut Syndrome, a rare form of childhood epilepsy

UCB, a global biopharmaceutical company, on Monday announced that FINTEPLA (fenfluramine) oral solution CIV has been approved in the United States, by the U.S....

Marinus Pharmaceuticals has achieved FDA approval for its first and sole drug

Marinus Pharmaceuticals, Inc., a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, announced that the U.S. Food and Drug...

Emmes makes 4th acquisition in CRO focused on rare diseases

Emmes, a global, full-service Clinical Research Organization (CRO) dedicated to supporting the advancement of public health and biopharmaceutical innovation, announced that it has acquired...

illumina launches multiple-gene test to spot rare treatable cancers

Illumina, Inc. today announced the launch of TruSight™ Oncology (TSO) Comprehensive (EU), a single test that assesses multiple tumor genes and biomarkers to reveal...

Ergomed launches Rare Disease Innovation Center

ERGOMED plc a leading Clinical Research Organization focused on rare disease drug development today announced the establishment of its Rare Disease Innovation Center™. Rare diseases...

EMA – New medicine for rare type of eye cancer

EMA has recommended granting a marketing authorisation in the European Union (EU) for Kimmtrak (tebentafusp), a monotherapy for the treatment of adult patients with...
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