Galsulfase

How it works

It supplies the deficient enzyme N-acetylgalactosamine-4-sulfatase, restoring degradation of dermatan sulfate and reducing glycosaminoglycan accumulation in tissues.

Prescribing in practice

• Infusion-related and anaphylactic reactions can occur, so administer under supervision with resuscitation facilities and appropriate pre-treatment available.
• It should be prescribed and supervised by a clinician experienced in managing lysosomal storage disorders.
• Slow the infusion or interrupt it if reactions develop, following the SPC for management and re-challenge.

Monitoring

Monitor closely during and after each infusion for hypersensitivity and assess clinical response over time.

Counselling the patient

• Infusions are given regularly and require monitoring for reactions such as rash, fever or breathing difficulty.
• Tell staff immediately if you feel unwell during an infusion.
• This treatment controls disease burden but does not cure the underlying condition.

Evidence & guidelines

Enzyme replacement for mucopolysaccharidosis type VI is an established orphan-disease therapy supported by controlled trial evidence.