Patients Zebras, not horses: combatting rare disease via education Elsevier launches open-access journal 'Rare' and short-form videos to educate on rare disease.
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.
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.
R&D Strategies in securing funding for rare disease research An outline of emerging financing strategies for rare disease research in challenging times.
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 Lily gets FDA nod for SERD-CDK 4/6 combo in breast cancer Eli Lilly's imlunestrant (Inluriyo) for breast cancer has received a new FDA approval, in combination with CDK4/6 inhibitor abemaciclib (Verzenio).
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