Learn About Mucopolysaccharidosis Type 4A

What is the definition of Mucopolysaccharidosis Type 4A?
Mucopolysaccharidosis type IVA (MPS IVA, also called Morquio syndrome, type A) is a metabolic condition that primarily affects the skeleton. The severity, age of onset, and associated symptoms vary significantly from person to person and range from a severe and rapidly progressive, early-onset form to a slowly progressive, later-onset form. The severe form is usually diagnosed between ages 1 and 3, while the milder form may not become evident until late childhood or adolescence. Signs and symptoms include various skeletal abnormalities such as short stature, knock knees, pectus carinatum, and malformations of the spine, hips and wrists. Affected people may also experience involvement of other organ systems such as respiratory problems, valvular heart disease, hearing impairment, corneal clouding, dental abnormalities, hepatomegaly, and spinal cord compression. MPS IVA is caused by changes (mutations) in the GALNS gene and is inherited in an autosomal recessive manner. Treatment is based on the signs and symptoms present in each person.
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What are the alternative names for Mucopolysaccharidosis Type 4A?
  • MPS IVA
  • MPS 4A
  • Morquio A disease
  • Galactosamine-6-sulfatase deficiency
  • GALNS deficiency
  • Morquio disease type A
  • MPS4A
  • MPSIVA
  • Mucopolysaccharidosis type 4A
  • N-acetylgalactosamine-6-sulfate sulfatase deficiency
  • Morquio syndrome A
Who are the top Mucopolysaccharidosis Type 4A Local Doctors?
Elite
Highly rated in
15
conditions

University Of Pretoria

Salford, ENG, GB 

Christian Hendriksz is in Salford, United Kingdom. Hendriksz is rated as an Elite expert by MediFind in the treatment of Mucopolysaccharidosis Type 4A. He is also highly rated in 15 other conditions, according to our data. His top areas of expertise are Mucopolysaccharidosis Type 4A, Mucopolysaccharidosis Type 4, Brachydactyly Mononen Type, and Multiple Sulfatase Deficiency.

Elite
Highly rated in
25
conditions
Gastroenterology
Pediatric Gastroenterology

UCSF Benioff Children’s Hospital Oakland

Oakland, CA 

Paul Harmatz is a Gastroenterologist and a Pediatric Gastroenterologist in Oakland, California. Dr. Harmatz has been practicing medicine for over 46 years and is rated as an Elite doctor by MediFind in the treatment of Mucopolysaccharidosis Type 4A. He is also highly rated in 25 other conditions, according to our data. His top areas of expertise are Mucopolysaccharidosis Type 6, Mucopolysaccharidosis Type 2, Mucopolysaccharidosis Type 4A, and Mucopolysaccharidosis Type 4. He is licensed to treat patients in California.

 
 
 
 
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Distinguished
Highly rated in
9
conditions

Pontificia Universidad Javeriana

Bogota, DC, CO 

Carlos Diaz-Almeciga is in Bogota, Colombia. Diaz-Almeciga is rated as a Distinguished expert by MediFind in the treatment of Mucopolysaccharidosis Type 4A. He is also highly rated in 9 other conditions, according to our data. His top areas of expertise are Mucopolysaccharidosis Type 4, Mucopolysaccharidosis Type 4A, Brachydactyly Mononen Type, and Pectus Carinatum.

What are the latest Mucopolysaccharidosis Type 4A Clinical Trials?
A Randomized Clinical Trial to Evaluate the Effects of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI
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Longitudinal Study of Neurodegenerative Disorders
What are the Latest Advances for Mucopolysaccharidosis Type 4A?
Impact of long-term elosulfase alfa treatment on clinical and patient-reported outcomes in patients with mucopolysaccharidosis type IVA: results from a Managed Access Agreement in England.
Diagnostic journey and impact of enzyme replacement therapy for mucopolysaccharidosis IVA: a sibling control study.
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Adult Morquio syndrome requiring occipito-thoracic fusion.