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PWS Outcomes Assessment Study

2019-09-30 07:06:56 | BioPortfolio

Summary

This is a longitudinal study during which qualitative interviews will be conducted with caregivers of Soleno C601/C602 study subjects. There is an additional option for caregivers to collect video data of PWS patients doing specific activities of daily life.

The purpose of this study is to understand the real-world and nuanced impact of a potential therapeutic on individual PWS patients. The results of this study will complement the outcomes being captured during the Soleno C601/C602 clinical studies.

There is no treatment or intervention associated with this study.

Study Design

Conditions

Prader-Willi Syndrome

Intervention

Interview

Location

Casimir Trials
Plymouth
Massachusetts
United States
02360

Status

Recruiting

Source

Soleno Therapeutics, Inc.

Results (where available)

View Results

Links

Published on BioPortfolio: 2019-09-30T07:06:56-0400

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Medical and Biotech [MESH] Definitions

An autosomal dominant disorder caused by deletion of the proximal long arm of the paternal chromosome 15 (15q11-q13) or by inheritance of both of the pair of chromosomes 15 from the mother (UNIPARENTAL DISOMY) which are imprinted (GENETIC IMPRINTING) and hence silenced. Clinical manifestations include MENTAL RETARDATION; MUSCULAR HYPOTONIA; HYPERPHAGIA; OBESITY; short stature; HYPOGONADISM; STRABISMUS; and HYPERSOMNOLENCE. (Menkes, Textbook of Child Neurology, 5th ed, p229)

A directed conversation aimed at eliciting information for psychiatric diagnosis, evaluation, treatment planning, etc. The interview may be conducted by a social worker or psychologist.

Standardized clinical interview used to assess current psychopathology by scaling patient responses to the questions.

Work consisting of a conversation with an individual regarding his or her background and other personal and professional details, opinions on specific subjects posed by the interviewer, etc.

Condition with a variable constellation of phenotypes due to deletion polymorphisms at chromosome location 22q11. It encompasses several syndromes with overlapping abnormalities including the DIGEORGE SYNDROME, VELOCARDIOFACIAL SYNDROME, and CONOTRUNCAL AMOMALY FACE SYNDROME. In addition, variable developmental problems and schizoid features are also associated with this syndrome. (From BMC Med Genet. 2009 Feb 25;10:16) Not all deletions at 22q11 result in the 22q11deletion syndrome.

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