FDA clears Marinus’ Ztalmy as first drug for rare epilepsy
epilepsy, ganaxolone, Marinus Pharma, rare disease, regulatory approval, Ztalmy
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rare disease
Amplifying the voices in rare diseases
Axon, Deep Dive, Deep Dive: Research and Development, Dr Emma Lemon, rare disease, research and development
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Illumina debuts its multi-gene test for cancer in Europe
diagnosis, genome, Illumina, Oncology, rare disease, TruSight Oncology
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FDA turns down Amryt’s epidermolysis bullosa drug
Amryt Pharma, dermatology, epidermolysis bullosa, inherited disease, rare disease, regulatory
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TargED Bio raises €39m for new clot-busting drug
acquired thrombotic thrombocytopenic purpura, cardiovascular, rare disease, stroke, TargED Biopharmaceuticals, thrombolysis
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Regenxbio expands MPS I gene therapy trial after first look at data
gene therapy, Hurler syndrome, rare disease, Regenxbio
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Astellas says Pompe gene therapy clears safety hurdle
Astellas, gene therapy, patients, Pompe disease, rare disease, safety
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