We are focused on treating rare, inherited disorders where the disease burden on children, families and caregivers is immense, and where current therapeutic options often do not exist, are suboptimal or are associated with severe complications. Read more about our current portfolio of products below.
Libmeldy is indicated for the treatment of metachromatic leukodystrophy (MLD) characterized by biallelic mutations in the arylsulfatase A (ARSA) gene leading to a reduction of the ARSA enzymatic activity:
– in children with late infantile or early juvenile forms, without clinical manifestations of the disease,
– in children with the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline.
For full information on this product, please check the links below.
Strimvelis is indicated for the treatment of patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available. For full information on this product, please check the links below.
Reporting of side effects: If you get any side effects, talk to your doctor, pharmacist, or nurse. This includes any possible side effects not listed in the package leaflet. You can also report side effects directly via the Yellow Card Scheme at https://yellowcard.mhra.gov.uk/. By reporting side effects, you can help provide more information on the safety of this medicine.
Adverse events should also be reported to email@example.com.
IE-002-2200006, July 2022