rare diseases

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Pharma

Mirum scores FDA approval for ultra-rare disorder

Rare disease pharma Mirum scored an approval for its ultra-rare bone lesion treatment it bought from Incyte.
Donavyn Coffey Sep 25, 2026 4:10pm
eric dube

Travere CEO Eric Dube to step down

Sep 22, 2026 10:10am
Sunrise over foggy forest in Tuscany Italy

FDA approves Ultragenyx gene therapy after prior rejection

Sep 17, 2026 4:50pm
Ionis headquarters

Ionis scores ‘breakthrough’ rare disease approval

Sep 4, 2026 11:18am
Lisraya Roivant Priovant

Priovant secures 1st FDA nod in rare skin and muscle disease

Aug 27, 2026 9:30pm
Boehringer Ingelheim booth at AACR 2026

Boehringer again rises up rare disease reputation rankings

Aug 20, 2026 4:29am