Methods of treating prader willi syndrome and conditions associated with low basal metabolic rate or hyperphagia using a katp channel opener

a technology of basal metabolic rate and katp channel, which is applied in the direction of drug composition, peptide/protein ingredients, anhydride/acid/halide active ingredients, etc., can solve the problems of undesirable side effects of many of the aforementioned drugs currently used to treat pws

Inactive Publication Date: 2014-12-11
SEDOGEN
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Benefits of technology

[0007]Further provided herein is a method for treating Prader-Willi Syndrome which may comprise administering a KATP channel opener to a patient in need thereof. The patient may be in PWS nutritional phase 0, 1a, 1b, 2a, 2b, 3, or 4. The patient's PWS nutritional phase may be prevented from progressing to a later phase. The patient may be in PWS nutritional phase 1a, 1b, 2a, or 2b, and the patient's PWS nutritional phase may be prevented from progressing to phase 1b, 2a, 2b, or 3. The patient's PWS nutritional phase may be shifted back to an earlier PWS nutritional phase. The patient may be in PWS nutritional phase 3 or 4, and the patient's PWS nutritional phase may be shifted back to phase 1a, 1b, 2a, or 2b.

Problems solved by technology

Many of the aforementioned drugs that are currently used to treat PWS have undesirable side effects.

Method used

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  • Methods of treating prader willi syndrome and conditions associated with low basal metabolic rate or hyperphagia using a katp channel opener
  • Methods of treating prader willi syndrome and conditions associated with low basal metabolic rate or hyperphagia using a katp channel opener
  • Methods of treating prader willi syndrome and conditions associated with low basal metabolic rate or hyperphagia using a katp channel opener

Examples

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Effect test

example 1

Treating PWS

[0157]This example shows how a KATP channel opener addresses abnormalities observed in patients with PWS. In particular, the channel opener diazoxide may hyperpolarize hypothalamic neurons in a manner similar to leptin.

[0158]Diazoxide has now been prescribed at low doses for an infant with PWS who had experienced hypoglycemic episodes. The infant was in PWS nutritional phase 1a transitioning to phase 1b was started at 0.5 mg / kg BID of diazoxide at eight months of age, increasing to 1 mg / kg BID. No changes in blood pressure or heart rate were observed, and no signs of edema were present. Within two weeks of starting diazoxide, the infant's strong narcoleptic response to solid foods had been virtually eliminated. Subjectively, her energy level and stamina also improved, and her therapists documented her significant progress. The infant's baseline IGF-1 levels were 152 ng / mL while on 0.8 mg QD of Genotropin. Around three weeks after starting diazoxide levels, the IGF-1 leve...

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Abstract

This invention relates to treating Prader-Willi Syndrome (PWS) using a KATP channel opener. The channel opener may be coadministered with other therapies used to treat PWS, such as human growth hormone, a wakefulness promoting agent, or a psychiatric or mood stabilizing drug, thereby allowing the baseline dosages of these other therapies to be decreased or making these other therapies unnecessary. The invention also relates to treating PWS based on the PWS nutritional phase of a patient, to prevent the patient's PWS nutritional phase from progressing or shift the patient's PWS nutritional phase back to an earlier phase. The invention further relates to treating PWS, and conditions associated with low basal metabolic rate or hyperphagia, with the KATP channel opener based on a patient's blood ketone levels.

Description

FIELD OF THE INVENTION[0001]This invention relates to treating Prader-Willi Syndrome (PWS) and conditions affecting basal metabolic rate using a KATP channel opener. The KATP channel opener may be coadministered with other therapies used to treat PWS, such as human growth hormone, a wakefulness promoting agent, or a psychiatric / mood stabilizing medication, which may allow the dose of these other therapies to be reduced relative to the dose that would otherwise be given to a patient with PWS in the absence of the KATP channel opener (e.g., baseline dose). The KATP channel opener may also obviate the need for the use of the other therapies used to treat PWS, such that PWS may be treated without human growth hormone, a wakefulness promoting agent, or a psychiatric / mood stabilizing medication. The KATP channel opener dosage may also be administered according to the PWS nutritional phase of a patient. The KATP channel opener may also be administered to prevent the PWS patient's PWS nutri...

Claims

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Application Information

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Patent Type & Authority Applications(United States)
IPC IPC(8): A61K38/27A61K31/549
CPCA61K31/549A61K38/27A61K31/145A61K31/19A61K31/198A61K31/522A61K45/06A61K38/095A61K2300/00
Inventor COTTER, SARA PAIGE
Owner SEDOGEN
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