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Dr. Andrew Rynne
MD
Dr. Andrew Rynne

Family Physician

Exp 50 years

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Suggest Remedy For Congestion Due To Niemann's Pick C Disease

My 34 years old son is a NIEMANN S PICK C DISEASE( neuro degenerative) having continually profuse, viscid, tenacious sticky mucus, obstructing breathing & getting accumulated in food pipe causing dysphagia.Asthalin & budate neubilisation though have given a little relief yet are not free from inherent side effects of these respules & management is difficult as he is hardly able to walk. What can you suggest to ease the situation?
Thu, 25 Jun 2015
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Pathologist and Microbiologist,  's  Response
Hi. There is a medication approved for Niemann Pick C called Miglustat. This has shown efficacy according to the evidence based literature. This medication works by inhibiting an enzyme which therefore reduces the buildup of the problematic lipid substance in the cells. That's a simple way of saying it. Dosage given in some studies is 200 mg orally thrice a day. Effect takes time but seems to be OK. This medicine should be prescribed and taken only if your physician tells you to do so because there are side effects and interactions. Monitoring is also necessary. Best regards.
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Suggest Remedy For Congestion Due To Niemann's Pick C Disease

Hi. There is a medication approved for Niemann Pick C called Miglustat. This has shown efficacy according to the evidence based literature. This medication works by inhibiting an enzyme which therefore reduces the buildup of the problematic lipid substance in the cells. That s a simple way of saying it. Dosage given in some studies is 200 mg orally thrice a day. Effect takes time but seems to be OK. This medicine should be prescribed and taken only if your physician tells you to do so because there are side effects and interactions. Monitoring is also necessary. Best regards.