Rare Disease

Showing all 13 results

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  AGAMREE (vamorolone)

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  CABLIVI (caplacizumab-yhdp) for injection

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  DUVYZAT (givinostat) oral suspension

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  FABHALTA (iptacopan)

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  FILSUVEZ (birch triterpenes)

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  KISUNLA (donanemab-azbt) injection

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  OJEMDA (tovorafenib) tablets

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  POMBILITI (cipaglucosidase alfa-atga)

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  RIVFLOZA (nedosiran)

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  RYSTIGGO (rozanolixizumab-noli)

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  RYZNEUTA (efbemalenograstim alfa-vuxw)

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  VEOPOZ (pozelimab-bbfg)

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  VORANIGO (vorasidenib) tablets

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