This expanded access program provides access to ulefnersen for eligible patients with FUS-associated amyotrophic lateral sclerosis (FUS-ALS) who meet protocol-defined eligibility criteria. Access is intended from the availability of FUSION clinical trial results until commercial availability of ulefnersen in participating countries. Ulefnersen is provided free of charge.
Ability to provide written informed consent (and assent, when required based on age and local requirements) before participation.
Confirmed disease-causing (pathogenic) variant in the FUS gene, verified by an approved genetic testing laboratory and reviewed by the program geneticist.
Age 10 years or older, with test results, signs, or symptoms showing ongoing loss of motor nerve cells consistent with amyotrophic lateral sclerosis (ALS).
Medical ability to undergo repeated injections into the fluid surrounding the spinal cord and other program-related procedures.
Any active or ongoing medical condition that, in the opinion of the treating physician, may increase the risk of treatment with ulefnersen.
Any medical condition or circumstance that makes repeated spinal taps (lumbar punctures) unsafe or unsuitable.
Expected need for treatment with blood-thinning medicines (anticoagulants) or medicines that reduce blood clotting (antiplatelet medications) during the program, as determined by the treating physician.
Ulefnersen
DrugAntisense oligonucleotide solution for intrathecal injection.
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