Sales & Marketing Delivering for the 5 in 10,000 patients with a rare disease An estimated 30 million people are living with a rare disease in the European Union alone.
News Phase 3 trial win sets up rare disease drug filing for CSL VANUGUARD trial of garadacimab in HAE sets up a pursuit of Takeda's fast-growing Takhyzro.
R&D Strategies in securing funding for rare disease research An outline of emerging financing strategies for rare disease research in challenging times.
News Regeneron is off to CHAPLE as FDA starts pozelimab review The C5 inhibitor is vying to become the first approved drug for the ultra-rare immune disorder.
News Travere undercuts key rival in rare kidney disease IgAN Filspari will launch for IgA Nepropathy at a discount to Calliditas' Tarpeyo, cleared in 2021.
News FDA gives green light to Chiesi's Lamzede Chiesi scores milestone US FDA approval for first AM enzyme replacement therapy, Lamzede.
News Remepy's hybrid Parkinson's therapy poised for phase 3 Remepy's Hybridopa therapy for Parkinson's, which combines an app with levodopa therapy, has shown efficacy in a phase 2 pilot study.
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