rare disease
Deep Dive/ R&D/ Views & Analysis
Amplifying the voices in rare diseases
mike.hammerton@pharmaphorum.com
Axon, Deep Dive, Deep Dive: Research and Development, Dr Emma Lemon, rare disease, research and development
0 Comment
Illumina debuts its multi-gene test for cancer in Europe
Phil Taylor
diagnosis, genome, Illumina, Oncology, rare disease, TruSight Oncology
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FDA turns down Amryt’s epidermolysis bullosa drug
Phil Taylor
Amryt Pharma, dermatology, epidermolysis bullosa, inherited disease, rare disease, regulatory
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TargED Bio raises €39m for new clot-busting drug
Phil Taylor
acquired thrombotic thrombocytopenic purpura, cardiovascular, rare disease, stroke, TargED Biopharmaceuticals, thrombolysis
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Patients/ Views & Analysis/ Views and analysis
Empowering rare disease patient advocacy organisations with data
Jessica Hagen
CZI, Komodo Health, rare disease, Web Sun
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Regenxbio expands MPS I gene therapy trial after first look at data
Phil Taylor
gene therapy, Hurler syndrome, rare disease, Regenxbio
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Astellas says Pompe gene therapy clears safety hurdle
Phil Taylor
Astellas, gene therapy, patients, Pompe disease, rare disease, safety
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Sanofi claims FDA okay for drug to treat rare blood disorder CAD
Phil Taylor
cold agglutinin disease, Enjaymo, fda, rare disease, regulatory approval, Sanofi, sutimlimab
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