News FDA clears first therapy for rare disease CTX from Mirum Mirum Pharma's Ctexli has become the first FDA-approved therapy for ultra-rare disease cerebrotendinous xanthomatosis (CTX).
News Can Dupixent plug treatment gap in bullous pemphigoid? The FDA is scheduled to deliver a verdict on Sanofi and Regeneron's Dupixent for rare autoimmune skin disorder bullous pemphigoid in June.
News EU approves CSL's once-monthly HAE drug Andembry The EU has approved CSL's once-monthly Andembry for hereditary angioedema, challenging therapies from Takeda and Biocryst.
News With Merck circling, SpringWorks gets new drug approval Merck KGaA is talking to SpringWorks about a takeover, and FDA approval for a neurofibromatosis drug could have sweetened any potential deal.
News Storybook can help kids understand rare kidney disease An e-Book aimed at children with rare kidney disease aHUS can help them understand the condition, deal with emotions, and find support, says Alexion.
R&D Life Sciences Industry Report - Rare Disease Space There are some 7,000 known rare diseases in the world, yet, multiple challenges and unmet needs remain when it comes to therapies for these diseases.
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