Prader-Willi Syndrome and its causes
Prader-Willi Syndrome (PWS) is a genetic disorder that occurs in infants with genomic imprinting. Genomic imprinting is a phenomenon in which certain genes are expressed as a copy of the gene inherited from one of the parent. Therefore, if the maternal copy of the gene is silenced, then, the paternal becomes active and vice versa. In most cases of PWS, the paternal copy of a segment of chromosome 15 gets deleted while the maternal copy remains inactive while in some cases, the infant receives only one maternal copy instead of inheriting one copy from each parent. In other cases, a mutation or defect that makes the chromosome 15 inactive can also cause Prader-Willi Syndrome.
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Though the disorder is as rare as affecting 1 out of 10,000 to 30,000 people, the health conditions associated with it draws serious concerns. During infancy, weak muscle, feeding issues and slow growth and development occur. During childhood, symptoms of overeating leading to obesity, sleep apnea and diabetes mellitus is common. Besides, behavioural problems like stubbornness, temper outbursts, compulsive behaviour are also observed. Distinct facial features like unusually fair skin, red hair, short stature, small hands and feet are seen besides mental problems like learning and intellectual disabilities. Children affected with this disorder are usually infertile.
PWS is an early embryonic defect and therefore, it is not linked to inheritance. However, in certain rare cases, a defective gene might be passed on causing genomic imprinting and leading to Prader-Willi Syndrome. Though incurable, by administering certain growth hormones and medication besides putting a check on the diet of the patient can help reduce the symptoms and health conditions associated with PWS. For the mental issues, parental training which guides them to deal with the children suffering from PWS has been proved helpful. In certain PWS related conditions, undergoing a surgical procedure might also be necessary.
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Global Markets Direct’s Pharmaceutical and Healthcare latest pipeline guide Prader-Willi Syndrome (PWS) – Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Prader-Willi Syndrome (PWS) (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.
The Prader-Willi Syndrome (PWS) (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Prader-Willi Syndrome (PWS) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 5, 1, 4 and 1 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 1 molecules, respectively.
Prader-Willi Syndrome (PWS) (Genetic Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.
Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
– The pipeline guide provides a snapshot of the global therapeutic landscape of Prader-Willi Syndrome (PWS) (Genetic Disorders).
– The pipeline guide reviews pipeline therapeutics for Prader-Willi Syndrome (PWS) (Genetic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
– The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
– The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
– The pipeline guide reviews key companies involved in Prader-Willi Syndrome (PWS) (Genetic Disorders) therapeutics and enlists all their major and minor projects.
– The pipeline guide evaluates Prader-Willi Syndrome (PWS) (Genetic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
– The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
– The pipeline guide reviews latest news related to pipeline therapeutics for Prader-Willi Syndrome (PWS) (Genetic Disorders)
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