Prognosis is a horizon scanning service that monitors emerging health technologies as they go through clinical trial development and the regulatory approval process.
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Galafold is a first-in-class oral chaperone therapy for treatment of Fabry disease in patients with amenable GLA gene variants. Migalastat works by stabilizing specific mutant forms of α-galactosidase, increasing enzyme trafficking to lysosomes. A proprietary in vitro assay Is used to classify GLA mutations as “amenable” or “not amenable” to treatment with migalastat.
Onpattro (patisiran) is an intravenous RNA interference (RNAi) therapeutic agent for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. RNAi is a natural cellular process of gene silencing. Onpattro is designed to silence specific messenger RNA, potentially blocking the production of TTR protein before it is made. This may help to enable the clearance of TTR amyloid deposits in peripheral tissues and potentially restore function to these tissues.
Poteligeo (mogamulizumab-kpkc) is an intravenously administered, humanized immunoglobulin G1 monoclonal antibody indicated for the treatment of patients with mycosis fungoides or Sézary syndrome who have failed to adequately respond to at least 1 systemic therapy. Poteligeo targets CC chemokine receptor 4 (CCR4), a novel target for immunotherapy.
Azedra (iobenguane I 131) is a radiotherapeutic product for treatment of adult and pediatric patients 12 years of age and older with iobenguane scan positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma (PPGL) who require systemic anticancer therapy. Azedra is the first drug approved to treat patients with PPGL who require systemic treatment.
The Early Bird Bleed Monitor System (EBBMS) is comprised of a standard vascular access sheath with embedded sensors that measure the electrical resistance across the blood vessel. By sensing a change in the vessel’s electrical resistance, the EBBMS is designed to detect and monitor bleeding from a blood vessel accidentally injured during endovascular procedures. When there is a vessel injury at the femoral access site, blood begins to accumulate outside the vessel, causing a change in impedance; a user interface display alerts the user to the change in impedance and a potential bleeding complication. The EBBMS is sensitive enough to...