Are there any treatments for Morquio syndrome?
All children need monitoring. Orthopedic conditions like scoliosis, kyphosis, and leg deformities affect many children with Morquio syndrome and may require surgical intervention. A medication has been developed to help in the treatment of Morquio, but currently there is no cure.
Are the treatments for Morquio syndrome successful?
Currently, there is no effective therapy for established skeletal abnormality in patients with Morquio A. Thus, patients often require multiple orthopedic surgeries in the upper cervical spine and lower extremities in their first decade of life [7].
What are the 2 types of Morquio syndrome?
Classification. This syndrome has two forms, A and B, referred to as Morquio A and Morquio B syndrome or MPS IVA and MPS IVB. The two forms are distinguished by the gene product involved; Type A involves a malfunction in the GALNS gene, while Type B involves a malfunction of the GLB1 gene.
What is the outlook for Morquio syndrome?
Outlook. Long-term outcomes for children with Morquio syndrome vary from child to child. Most children with Morquio syndrome have normal intelligence. Children with spine and bone conditions can have difficulty walking and may require a wheelchair; others may be more mobile.
How long is enzyme replacement therapy?
(This compensates for the missing enzyme, which is why the therapy is called enzyme replacement.) Patients receive ERT via IV infusion, which usually takes 1 to 2 hours.
How much does enzyme replacement cost?
Paying for Enzyme Replacement Therapy ERT can be very expensive, totaling up to $200,000 or more each year. Insurance may cover most of this cost, and resources are available if you need help paying for ERT treatments.
What is Morquio syndrome Type B?
Abstract. Morquio B disease (MBD) is an autosomal recessive GLB1-gene-related lysosomal storage disease, presenting with a peculiar type of dysostosis multiplex which is also observed in GALNS-related Morquio A disease.
Is there an enzyme replacement therapy for Morquio A?
Hendriksz CJ, Burton B, Fleming TR, et al. Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study. J Inherit Metab Dis. 2014;37 (6):979-990.
How is ERT used to treat Morquio A?
When Morquio A patients are treated by ERT, it is likely that urine KS level is reduced rapidly since the enzyme is delivered to kidney and digests the KS stored in kidney.
What is the cause of Morquio A syndrome?
Morquio A syndrome is an autosomal recessive disorder, one of 50 lysosomal storage diseases (LSDs), and is caused by the deficiency of N-acetylgalactosamine-6-sulfate sulfatase (GALNS). Deficiency of this enzyme causes specific glycosaminoglycan (GAG) accumulation: keratan sulfate (KS) and chondroitin-6-sulfate (C6S).
Is there a Morquio registry for MPS IVA?
There is a Morquio A Registry that collects data on pregnant women and nursing mothers with MPS IVA who are treated with VIMIZIM. Contact [email protected] for information and enrollment. Safety and effectiveness in pediatric patients below 5 years of age have not been established and are currently being evaluated.