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Alexion, AstraZeneca Rare Disease, is the group within AstraZeneca focused on rare diseases, created following the 2021 acquisition of Alexion Pharmaceuticals, Inc. As a leader in rare diseases for 30 years, Alexion is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialization of life-changing medicines. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on hematology, nephrology, neurology, metabolic disorders, cardiology and ophthalmology. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries.
Guided by our purpose, we work As One for Patients to take a holistic approach to healthcare, designing medicines and intelligent devices that support patients throughout their care journey. Our work spans oncology, rare tumors - including NF1-PN, desmoid tumors, and tenosynovial giant cell tumor (TGCT), multiple sclerosis, and fertility, alongside digital tools and biomarker-driven approaches that help personalize care. In rare tumors, we are on a mission to improve patient lives, grounded in urgency and scientific rigor, to focus on outcomes that matter most to patients and impact their realities.
Pasithea Therapeutics is a clinical-stage biotechnology company developing PAS-004, a next-generation macrocyclic oral MEK inhibitor, for the long-term treatment of chronic diseases including the neurocutaneous manifestations of neurofibromatosis type 1 (NF1). Pasithea is currently testing PAS-004 in a Phase 1 clinical trial in advanced cancer patients (NCT06299839), and a Phase 1/1b clinical trial in adult patients with neurofibromatosis type 1 (NF1)-associated plexiform neurofibromas (NCT06961565).
The 2026 European Neurofibromatosis Conference in Vienna (12–14 November) is now fully booked. Explore advances in NF1, Schwannomatosis, and patient-centred research at Hyatt Regency Vienna (previously Andaz Vienna Am Belvedere).