Is Anyone Familar With Mps 3 and the Drug Pamidronate?
Question by bonna l: is anyone familar with mps 3 and the drug pamidronate?
my grandaughter has mp3 and it is very apparent she is getting stiffer injoints ,she swims and has theraphy.rides bike now at the age of ten,but just seems no dr in columbus really has any knowledge of the benefits or the disadvantages of this drug she is on growth hormone and has gained 2inches in the last yr but her weight gain is still very lowjust hope someone out there can help ,thank you
Best answer:
Answer by cardiophile
A lot of information on pamidronate is available at this link:
http://www.mimsonline.com/Page.aspx?menuid=mng&name=pamidronate&h=pamidronate
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The mucopolysaccharidoses (MPSs) are a rare group of inherited lysosomal storage disorders that are caused by the deficiency or absence of specific lysosomal enzymes. The absence of these enzymes allows for the accumulation of complex carbohydrates in the body’s cells and tissues and in the cellular organelles, the lysosomes. These complex carbohydrates are known as mucopolysaccharides or glycosaminoglycans (GAGs) and serve as the building blocks for connective tissues in the body.
The MPSs comprise a group of 7 metabolic disorders, known as MPS types I-VII.
MPS III, or Sanfilippo syndrome, is the deficiency or absence of 4 different enzymes that are necessary to degrade the GAG heparan sulfate. Each enzyme deficiency defines a different form of Sanfilippo syndrome, as follows: type IIIA (Sanfilippo A), type IIIB (Sanfilippo B), type IIIC (Sanfilippo C), and type IIID (Sanfilippo D). However, severe mental retardation also occurs and is usually associated with this syndrome.
In terms of treatment, currently there is no cure for this disorder and it remains largely supportive. The behavioural disturbances of MPS-III respond poorly to medication. At the moment, Gene Therapy to treat MPS-III have been very promising in clinical trails and is under investigation for MPS-III in animal models.
Speaking of Pamidronate, it is an osteoporosis drug. This drug maintains bone strength, prevents fractures, and improves bone-pain. However, in long-term treatment, the kidney function may be harmed.
Overall, make sure your grand-daughter be given a proper diet and she be physically active. Usually, weight gain is normal in cases of MPS. Also, routinely health related check-ups are important for her, yourself and everyone else in your family.
Good Health To All Of You !