Tarui Disease

Debrancher Deficiency

GYG1 DEFICIENCY

Daily treatment with Triheptanoin oil for 14 days (7 days titration period in addition to 7 days full dose period with 1g/kg/day).

Triheptanoin

Daily treatment with Safflower oil for 14 days (7 days titration period in addition to 7 days full dose period with 1g/kg/day).

Placebo Oil

Copenhagen Neuromuscular Center

The Effect of Triheptanoin on Fatty Acid Oxidation and Exercise Tolerance in Patients With Glycogenoses

Triheptanoin's Effect on Fatty Acid Oxidation and Exercise Tolerance in Patients With Debrancher Deficiency, Glycogenin-1 Deficiency and Phosphofructoinase Deficiency at Rest and During Exercise. A Randomized, Double-blind, Placebo-controlled, Cross-over Study

Heart rate during constant load cycling exercise.

Palmitate oxidation measured via stable isotope technique and indiret calorimetry during constant load exercise.

The Short form-36 assesses eight health concepts: limitations in Quality of life physical activities because of health problems; limitations in social activities because of physical or emotional problems; limitations in usual role activities because of physical health problems); bodily pain; general mental health (psychological distress and well-being); limitations in usual role activities because of emotional problems; vitality (energy and fatigue); and general health perceptions. The standard form of the instruments asks for participants to reply to questions according to how they have felt over the previous week. The items use Likert-type scales, from 1-5 points, where 1 usually indicates that the patient is feeling worse.

Borg Score during constant workload cycling. The Borg RPE scale is a numerical scale that ranges from 6 to 20, where 6 means "no exertion at all" and 20 means "maximal exertion. The Borg scale is named after Borg GA 1982.

Bouchard's Physical Activity Record (BAR) is a widely used diary in which participants report physical activity for each 15 minute interval over three days. Activities are rated on a scale of 1 to 9 (1 = sedentary activity, 9 = intense manual work or high intensity sports) to yield a total energy expenditure score.

Mette Cathrine Oerngreen

Principal Investigator

Rigshospitalet, Denmark

Ultragenyx Pharmaceutical Inc

The study will be conducted as a randomized, placebo-controlled, double blind, crossover study consisting of two 14 days treatment periods set apart by minimum a 7-days wash-out period where no treatment is taken. In one treatment period, the subjects receive UX007 treatment and placebo treatment in the other. The study will consist of three separate groups; one group consisting of GSD III, one group of GSD VII, and one group of GSD XV subjects. Each of these groups will be blinded and randomized separately

Behavioral: Psychosocial stimulation, Cognitive behavioral therapy and positive parenting practice The mothers of intervention (ECD+HN) group will receive fortnightly group sessions for 12 months that will include combined messages on a) psychosocial stimulation, b) positive parenting to prevent child maltreatment and c) cognitive behavioural therapy (CBT) for positive thinking, d) health and nutrition messages and e) 15 micronutrient sprinkle supplement.(90 sachets of over 6 month-period)

Only regular health message from government health services

Maltreatment

The mothers of intervention (ECD+HN) group will receive fortnightly group sessions for 12 months that will include combined messages on a) psychosocial stimulation, b) positive parenting to prevent child maltreatment and c) cognitive behavioural therapy (CBT) for positive thinking, d) health and nutrition messages and e) 15 micronutrient sprinkle supplement.(90 sachets of over 6 month-period)

Psychosocial stimulation, Cognitive behavioral therapy and positive parenting practice

Runyan, Child-Maltreatment-Research-L (CMRL) list serve, 2014

Uddin MK, Islam MT, Asaduzzaman M. Perceived Stress and Self-Esteem as Significant Predictors of Cigarette Smoking Behavior of Bangladeshi Male Adults.

World Health Organization 2008, The Global Burden of Disease 2004 update

Fahmida Tofail, PhD

Reduce Childhood Maltreatment and Promote Development

Effect of Integrated Management of Childhood Maltreatment, Maternal Depression, Psychosocial Stimulation and Nutritional Intervention on Child Development - A Randomized Controlled Trial

cognitive development will be measured by Bayley tools. All individuals score will be aggregated in to composite score of cognitive,motor, language and social-emotional development of children

The International Society for the Prevention of Child Abuse and Neglect (ISPCAN) has developed ISPCAN Child Abuse Screening Tool- Parent Version (ICAST-P) will be used for measuring the severity of maltreatment calculating score.

Height will be measure in centimeter through World Health Organization (WHO) guideline.

Weight will be measure in gram through World Health Organization (WHO) guideline.

Head circumference will be measure in centimeter through World Health Organization (WHO) guideline.

The Hematological measurement will be measured as Hb per g/dl ,

folic acid status will be measured as ng/ml

The Hematological measurement will be measured as Retinol per Mg /ml

The Hematological measurement will be measured as STfR per Mg/ml

the Rosenberg Self Esteem Scale (Rosenberg, 1965), the most widely used measure of self esteem for research purposes and Measurement of maternal depressive symptoms will be done using Center for Epidemiologic Studies Depression Scale (CESD)

A questionnaire will be used to measure positive parenting

International Centre for Diarrhoeal Disease Research, Bangladesh

UBS Optimus Foundation

Systemic Lupus Erythematosus

Methotrexate and folic acid

University Health Network

Study of Methotrexate in Lupus Erythematosus

A Canadian Multicenter, Randomized, Double-Blind Placebo-Controlled Study of Methotrexate and Folic Acid in Systemic Lupus Erythematosus: A Phase III Trial.

riboflavin/fatigue

The Effect of Triheptanoin on Fatty Acid Oxidation and Exercise Tolerance in Patients With Glycogenoses

Reduce Childhood Maltreatment and Promote Development

Study of Methotrexate in Lupus Erythematosus

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