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Doctor insights on: Medicine For Pompe Disease

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How is pompe disease treated?

How is pompe disease treated?

Enzyme replacement: Through biotechnology, enzyme replacement has been developed, which should stabilize the symptoms. ...Read more

Dr. Amrita Dosanjh
7 Doctors shared insights

Pompe Disease (Definition)

Is glycogen storage disease type 2. Is an autosomal recessive metabolic disorder with muscle & nerve cell damage, leading to muscle weakness & ...Read more


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What exactly is pompe disease?

What exactly is pompe disease?

Glycogen storage: Pompes disease is a glygogen storage disorder type ii, due to lack of an enzyme glycogen (the way you store glucose in muscle and liver) can not be broken down. Glycogen excess in muscle causes severe muscle weakness (hypotonia) and eventuallybrespiratory failure (due to respiratory muscle involvement). ...Read more

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What is the definition of pompe disease?

GSD type II: This website will be helpful: http://www. Ninds. Nih. Gov/disorders/pompe/pompe. Htm. ...Read more

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Can you tell me how pompe disease in adults work?

Can you tell me how pompe disease in adults work?

Mutations of a gene: On Chromosome 17q25.3 that codes for a lysosomal enzyme that breaks down glycogen cause Glycogen Storage Disease Type II or Pompe Disease subtypes. It is autosomal recessive, the mutant gene comes from from both unaffected carrier parents. Age of onset & rate of progression of pelvic & leg, then arm & respiratory muscle weakness vary with subtype. See www. Agsdus. Org/html/typeiigsd. Html. ...Read more

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What is the definition or description of: pompe disease?

What is the definition or description of: pompe disease?

Pompe disease: Is glycogen storage disease type 2. Is an autosomal recessive metabolic disorder with muscle & nerve cell damage, leading to muscle weakness & myopathy throughout the body. ...Read more

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My grandson has infantile pompe disease and is presently being treated. I am in my sixties, should I go for testing?

My grandson has infantile pompe disease and is presently being treated. I am in my sixties, should I go for testing?

No: The chances of it making any difference now are negligible. You don't have the disease or you wouldn't live to be a grandfather. It is passed as a recessive gene through unaffected parents. Both parents must carry the recessive gene & could have normal, carrier or affected offspring. Other family members may want carrier testing but it is difficult & only available at specialty centers. ...Read more

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Do you know if adult pompe disease is caused by a sudden mutation or a long build up of glycogen?

Do you know if adult pompe disease is caused by a sudden mutation or a long build up of glycogen?

Long build up: I'm not a geneticist; and, there are good references on this subject easily available on the net. However, a quick answer is long build-up. The disease is viewed as constitutional (born-in) and not a later development of a "sudden mutation". If you are really interested, do your homework on the subject by looking at NIH and other sites. ...Read more

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What medicine I have to take for chrones disease?

Depends: You need to see a gastroenterologist for history & exam and guidance as to which of the options available is best for your situation. ...Read more

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For what disease is the drug aderall?

For what disease is the drug aderall?

Adderall (dextroamphetamine and racemic amphetamine): This drug is a stimulant. It is prescribed for add/adhd at times, and it is sometimes used for the fatigue of ms (multiple sclerosis) or after traumatic brain injury if monitored closely. ...Read more

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What medicines are use for lou gehrig's disease?

What medicines are use for lou gehrig's disease?

Treatment: It's mostly supportive, but a drug called Riluzole is approved for ALS and it's thought to work by decreasing glutamate mediated motor neuron cell death. ...Read more

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What is an experimental drug for Crohn's disease?

What is an experimental drug for Crohn's disease?

Clinical Trials: Although available, experimental drugs are given in clinical trials, usually in academic or private institutions with doctors involved in research, where patients that have failed to existing treatment are treat. There are a variety of immunotherapy medications like Remicade (infliximab) that are available for Crohn's you could discuss with your doctor about.
Ustekinumab is a clinical trial drug for Crohn's ...Read more

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What is the safest medicine name for peyronies disease?

What is the safest medicine name for peyronies disease?

Some injections: Non-surgical treatments for peyronie's include: waiting (as some penises improve without treatment), or injections of medications such as verapamil or interferon (medications that can decrease the collagen fibrous material in the plaque). An experienced urologist can evaluate to be sure peyronie's is cause of the curvature, and then he may recommend non-surgical treatments as a first step. ...Read more

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What are the diseases in which prednisolon medicine can be used at its best?

What are the diseases in which prednisolon medicine can be used at its best?

Several: Steroids can be used for back pain (inflammation), some brain/sinal cord injuries with swelling, asthma, COPD (emphysema), dermatitis (contact-posion ivy, etc), certain collagen vascular diseases, etc. Etc. Hope this helps. ...Read more

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Hi. What is the best drugs, pencillamin or trientine for wilson's disease?

Wilson's disease: Check out http://www. Mayoclinic. Com/health/wilsons-disease/ds00411 for info re wilson's disease which is autosomal recessive cause of excess copper. Both Penicillamine & trientine are used to chelate & remove excess copper. There's no best drug for everyone but rather a best drug for you. Talk to your doc about what's available to you, what's affordable, and what side effects are tolerable to you. ...Read more

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What drugs can I take for abnormal routine test? I fo not have a veneral disease.

Testing: Unfortunately, without knowing what part of your testing was abnormal, it is not possible to suggest a treatment. Please give some more specifics, or go see a doctor to review your test results. ...Read more

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Can you tell me an example of a time when medicine was shipped elsewhere to another country in order to stop an epidemic or disease?

Can you tell me an example of a time when medicine was shipped elsewhere to another country in order to stop an epidemic or disease?

Yes: The Canadian Health Minister indicated that Canada will donate between 800 to 1, 000 of their 1, 500 doses of an experimental Ebola Vaccine - VSV-EBOV to help with the Ebola-Zaire outbreak in West Africa. ...Read more

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How does nuclear medicine cure diseases? Can anyone give an example of how nuclear medicine is used to cure diseases? I don't understand how it works.

How does nuclear medicine cure diseases? Can anyone give an example of how nuclear medicine is used to cure diseases? I don't understand how it works.

The: The best example of how nuclear medicine is used to cure diseases is treatment of thyroid cancer or graves' disease (a form of hyperthyroidism). In both cases the goal is to destroy thyroid tissue. The thyroid gland contains a large amount of iodine which it uses to produce thyroid hormone. Once iodine is ingested, a large amount is concentrated within the thyroid gland. Nuclear medicine harnesses this principal to destroy thyroid tissue by giving the patient a radioactive form of iodine (i-131). This radioactive iodine is concentrated in the thyroid gland. The isotope primarily releases beta radiation which destroys the surrounding tissue (most of which is thyroid tissue). If there is thyroid cancer in the area, the cancer is also destroyed. ...Read more

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Hello, I'm 25 years, helthier, no diseases, 181cm high, 56kg weight, male, I'm too thin, I want a possible drug to increase my weight, my apetite is little.

Hello, I'm 25 years, helthier, no diseases, 181cm high, 56kg weight, male, I'm too thin, I want a possible drug to increase my weight, my apetite is little.

Drugs to gain weight: Are dangerous if no medical condition indicates their use. Better to eat higher protein such as eggs, dairy, etc. Exercise will tone up your body and help muscle mass. I'm glad your health is good. Enjoy that and focus on being a good person to others, neat and clean. Peace and good health. A smile goes a long way also. ...Read more

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My Father has NPH, Is any Medicine for this disease?

My Father has NPH, Is any Medicine for this disease?

You must mean: B PH (N PH is the name of a lind of Insulin) B-PH=BENIGN PROSTATIC HYPERTROPHY and YES there is medication for this disease as well as SURGICAL treatments!
Hope this helps!
Dr Z ...Read more

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What medicines help with degenerative disc disease?

What medicines help with degenerative disc disease?

A variety: Of non steroidal anti inflammatory medication as long as there are no contraindications like allergies, use of blood thinners, kidney or liver disease or ulcer/reflux issues. Tylenol (acetaminophen) is an option. Other types of drugs like cymbalta, Elavil and neurontin/lyrica may also help. Ideally, not smoking, regular exercise and weight control are also helpful. Good levels of vitamin d also are important. ...Read more

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Can coeliac disease prevent me from studying medicine?

Can coeliac disease prevent me from studying medicine?

No!!!!!: Many people live with celiac disease and are highly functioning professionals. Strict adherence to a gluten-free diet is crucial. That also means reading labels very carefully. You should shop at the gluten-free section, available in most groceries, be under competent medical supervision. Also see www. Celiac. Nih. Gov/materials. Aspx; www. Celiac. Org/ ...Read more

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What drug is used to treat hodgkin's disease lymphoma?

What drug is used to treat hodgkin's disease lymphoma?

ABVD: Adriamycin (doxorubicin)
Bleomycin
Vinbastine
Dacarbazine

The combination of the 4, all given every 2 weeks for 8 or 12 times depending on the stage of the cancer is the gold standard ...Read more

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I want the best medicine for removal of this disease ie milia?

I want the best medicine for removal of this disease ie milia?

Extraction: Milia are inclusion cysts. The best way to get rid of them is to have them extracted by an aesthetician or dermatology practice ...Read more

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Could you tell me what are some diseases brought by drug abuse?

Could you tell me what are some diseases brought by drug abuse?

Many: Drug or substance abuse can be at the helm of many disease states which can affect your mental, psychological, and physiological systems in your body. The list is long and includes every organ system causing accelerated or preventable damage to your body. ...Read more

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What over-the-counter medicine can be purchased for crons disease?

What over-the-counter medicine can be purchased for crons disease?

Depends: This depends in what type of symptoms you ae trying to treat. Over the counter medications should not be your only treatment for crohn's disease. ...Read more

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Why would immunosuppressant drugs make a patient susceptible to disease?

Why would immunosuppressant drugs make a patient susceptible to disease?

See below.: Immunosuppressive medications turn off the cells that are responsible for fighting infections. ...Read more