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Therapy Area

Rare Conditions.

Conditions we treat

  • Lysosomal storage disorders (Gaucher, Pompe, Fabry, Hunter, Hurler, MPS types)
  • Hereditary angioedema (HAE)
  • Paroxysmal nocturnal hemoglobinuria (PNH)
  • Atypical hemolytic uremic syndrome (aHUS)
  • Neuromyelitis optica spectrum disorder
  • Generalized myasthenia gravis
  • Pulmonary arterial hypertension (PAH)
  • Other ultra-rare metabolic and immunologic conditions

Rare disease therapies we deliver

TheraVite Health delivers and manages high-complexity, high-cost therapies for rare and ultra-rare conditions, including enzyme replacement therapy, complement inhibitors, and FcRn antagonists. Many of these therapies require specialized clinical monitoring and coordination with patients’ subspecialty physicians.

Infusion therapies (in-suite administration)

  • Cerezyme (imiglucerase), VPRIV (velaglucerase), Elelyso (taliglucerase) — ERT for Gaucher disease
  • Lumizyme (alglucosidase alfa), Nexviazyme (avalglucosidase alfa) — ERT for Pompe disease
  • Fabrazyme (agalsidase beta) — ERT for Fabry disease
  • Aldurazyme (laronidase), Naglazyme (galsulfase), Vimizim (elosulfase alfa), Mepsevii (vestronidase alfa) — ERT for MPS subtypes
  • Cinryze (C1 esterase inhibitor), Berinert (C1 esterase inhibitor) — HAE prophylaxis and acute treatment
  • Soliris (eculizumab), Ultomiris (ravulizumab) — IV C5 inhibitors for PNH and aHUS
  • Vyvgart (efgartigimod alfa) — IV FcRn antagonist for myasthenia gravis
  • Uplizna (inebilizumab) — IV anti-CD19 for NMOSD

Subcutaneous therapies (specialty pharmacy dispensing)

  • Vyvgart Hytrulo (efgartigimod alfa + hyaluronidase) — SC FcRn antagonist for myasthenia gravis
  • Firazyr (icatibant), Takhzyro (lanadelumab) — SC HAE therapies

Oral specialty medications

  • Galafold (migalastat) — oral pharmacological chaperone for Fabry disease
  • Orladeyo (berotralstat) — oral plasma kallikrein inhibitor for HAE prophylaxis

Pulmonary hypertension therapies

  • Tyvaso (treprostinil inhaled), Remodulin (treprostinil SC/IV), Veletri (epoprostenol IV), Flolan (epoprostenol IV)
TheraVite supports both administration (infusion) and dispensing (specialty pharmacy) for rare and ultra-rare conditions, with intensive clinical monitoring and care coordination across the full continuum of treatment.

What makes TheraVite’s approach distinct

Rare disease patients often face the highest stakes and the smallest margin for error. TheraVite’s clinical protocols are particularly suited to this — careful tracking of every dose, attentive monitoring of how you respond, and a dedicated pharmacist who knows your case in detail. Many rare disease therapies are also limited distribution drugs — TheraVite is pursuing manufacturer access on a drug-by-drug basis as URAC accreditation progresses. Contact us before referring or transferring a rare-disease patient to confirm current access for the specific therapy.

For referring providers

TheraVite handles prior authorization, benefits investigation, and payor credentialing for all therapies listed above. For drugs subject to limited distribution networks, we are pursuing manufacturer access in parallel with URAC accreditation. Contact us for current access status by drug.

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For patients

If you have been prescribed any of the therapies above, TheraVite can typically begin treatment within 5 to 10 business days of receiving your prescription. We handle insurance verification, prior authorization, and patient onboarding so you can focus on your therapy.

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