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BioWorld - Wednesday, April 29, 2026

Rare disease

Home » Topics » Disease categories and therapies » Rare disease
  • Eli lilly signage
    April 27, 2026
    By Karen Carey

    Lilly buying Ajax for up to $2.3B for next-gen JAK assets

    Eli Lilly and Co. is acquiring Ajax Therapeutics Inc. for up to $2.3 billion in cash, gaining access to next-generation JAK inhibitors for patients with myeloproliferative neoplasms.
  • Kidneys pills and chart line
    April 14, 2026
    By Karen Carey

    Travere’s Filspari approved for rare kidney disease FSGS

    Becoming the first and only fully FDA-approved treatment for focal segmental glomerulosclerosis (FSGS), Travere Therapeutics Inc.’s Filspari (sparsentan) has gained access to a second lucrative market with a regulatory package that validates proteinuria as a surrogate endpoint.
  • Knee joint giant cell tumor of bone
    April 13, 2026
    By Nuala Moran

    Synox en route to approval on positive phase III for TGCT

    Synox Therapeutics Ltd. is preparing to file for FDA approval of emactuzumab in treating tenosynovial giant cell tumor (TGCT), after announcing positive top-line phase III results.

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