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Rare Diseases Supported in France

In France, we work across various rare diseases and devastating conditions, including:  

Atypical Haemolytic Uraemic Syndrome (aHUS)

A rare disease that can cause progressive injury to vital organs, primarily the kidneys, via damage to the walls of blood vessels and blood clots.

Neurofibromatosis Type 1 (NF1) Plexiform Neurofibromas (PN)

A rare, progressive, genetic condition characterised by non-malignant (non-cancerous) tumors called plexiform neurofibromas (PN) that develop in the brain, and along the spinal cord and nerve.

Generalised Myasthenia Gravis (gMG)

A rare, autoimmune neuromuscular disease characterised by loss of muscle function and severe muscle weakness.

Neuromyelitis Optica Spectrum Disorder (NMOSD)

A rare autoimmune disease of the central nervous system, characterised by unpredictable relapses which can result in cumulative disability.

Hypophosphatasia (HPP)

A rare, genetic (inherited), metabolic disease characterised by impaired mineralisation (“calcification”), the process that hardens and strengthens bones and teeth. This can lead to poor growth and development, weakness and deformity of bones and other skeletal abnormalities, and premature loss of teeth with the root intact.

Lysosomal Acid Lipase Deficiency (LAL-D)

A genetic, and progressive ultra-rare metabolic disease associated with multiorgan damage in infant, paediatric and adult patients, and premature death in infants.

Medicines

Our medicines available in France: 

Kanuma

(sebelipase alfa) 2 mg/ml injection, for intravenous use

Koselugo

(selumetinib) 10 mg and 25 mg capsules, for oral use

Soliris

(eculizumab) 300 mg, injection, for intravenous use  

Strensiq

(asfotase alfa) 40 mg/ml and 100 mg/ml, injection, for subcutaneous use

Ultomiris

(ravulizumab) 100 mg/ml, injection, for intravenous use

Voydeya

(danicopan) 50 mg et 100 mg, film-coated tablets

Clinical Trials

Clinical Trials

Alexion is committed to serving people affected by rare diseases through the discovery, development and access of life-changing medicines. Our goal is to bring safe and effective medicines to as many patients as possible by conducting rigorous clinical trials and obtaining marketing approval by regulatory authorities around the world. Our work is marked by a commitment to the highest standards of preclinical and clinical research, and an acute sense of urgency. As such, we prioritize access to our investigational medicines through participation in a clinical trial.

Transparency and Disclosure

Careers

Career Opportunities

At Alexion, we foster an environment that attracts and retains the best talent. We provide the tools to enable each individual to perform at their personal best so that we maximize our collective impact.

France Office