Morquio Syndrome Drug Discovery Service
Creative Biolabs is well-known in the field of neuroscience and has completed many innovative projects in this field. Our advanced technology platform and years of project experience allow us to provide customers with a variety of products and services for morquio syndrome research.
Introduction to Morquio Syndrome
Morquio syndrome, also known as Mucopolysaccharidosis Type IV (MPS IV), is a rare recessive genetic condition that affects children's bones and spine, organs, and physical abilities. Children with morquio syndrome lack or cannot produce enough enzymes to break down the sugar chains that are naturally produced in the body. And these sugar chains gradually accumulate in cells, blood, tendons, and ligaments and cause damage to the body. The accumulation of these glycosaminoglycans, which cannot be processed by the body, in different parts of the body can cause symptoms in many different organ systems. Morquio syndrome is autosomal recessive, so both parents must carry the gene to pass it on to offspring.
Fig.1 Clinical manifestations of Morquio A disease. (Tomatsu, 2014)
Symptoms of Morquio Syndrome
The morquio syndrome can be divided into types A and B, while type B usually has milder symptoms. Type A relates to a malfunction in the GALNS gene, while type B relates to a malfunction of the GLB1 gene. Depending on the subtype, patients with Morquio syndrome live between 20 and 30 years. Causes of death mainly include respiratory failure, heart failure, post-traumatic organ failure, surgical complications, as well as a heart attack. The age of onset is usually between 1 and 3 years of age, and physical development stops around 8 years of age. This disease causes progressive changes to the ribs and thoracic bones and possible neurological complications. In addition, some patients may also have hearing loss and corneal opacities.
- Kyphosis or scoliosis
- Heart and vision problems
- Enlarged liver
- Knock knees
- Short height
Diagnostic and Treatment of Morquio Syndrome
Morquio syndrome can be diagnosed using different methods, including genetic testing, x-ray imaging, MRI scanning, echocardiogram, and laboratory testing. Prenatal identification and enzyme replacement therapy are effective treatments for morquio syndrome. Till now, there has been an FDA-approved drug for the treatment of type A. However, there is no treatment for type B. Based on our established animal models, we are willing to provide one-stop research services against Morquio syndrome.
- Orthopedic Surgical Procedures
- Enzyme Replacement Therapy (ERT)
- Hematopoietic Stem Cell Therapy (HSCT)
- Substrate Reduction Therapy (SRT)
- Gene Therapy
- Anti-inflammatory Drugs
Services for Morquio Syndrome
- In Vitro Services
- In Vivo Services
- Ex Vivo Services
- Discovery Services
- Development Services
Morquio Syndrome Drug Discovery Service
Equipped with world-leading platforms and extensive experience, Creative Biolabs provides one-stop drug discovery service for our clients. From the basic target identification to the final IND-enabling, we can meet all the needs of customers.
Creative Biolabs is a leading service provider that focuses on neuroscience research. We can assist you in designing the best research outline customized to meet the requirements of clients' programs. If you are interested in our services and products, please do not hesitate to contact us for more details.
Reference
- Tomatsu, S.; et al. Morquio A syndrome: diagnosis and current and future therapies. Pediatric endocrinology reviews: PER. 2014, 12(0 1): 141.
