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Therapies for rare diseases: therapeutic modalities, progress and challenges ahead

Nature reviews. Drug discovery, 2020-02, Vol.19 (2), p.93-111

2020

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Emotion regulation group skills training for adolescents and parents: A pilot study of an add-on treatment in a clinical setting

Clinical child psychology and psychiatry, 2020-01, Vol.25 (1), p.141-155

2020

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What Is Rare and What Is Orphan? A Guide to the Regulatory Terminology

Clinical pharmacology and therapeutics, 2024-09

Larsson, Kristina

2024

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Recommendations for the design of small population clinical trials

Orphanet journal of rare diseases, 2018-11, Vol.13 (1), p.195-195, Article 195

2018

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A Randomized Controlled Clinical Trial of Transanal Irrigation Versus Conservative Treatment in Patients With Low Anterior Resection Syndrome After Rectal Cancer Surgery

Annals of surgery, 2023-01, Vol.277 (1), p.30-37

2023

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Predictors associated with an increase in daily steps among people with prediabetes or type 2 diabetes participating in a two-year pedometer intervention

BMC public health, 2024-05, Vol.24 (1), p.1290-1290, Article 1290

2024

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Autonomous shuttles contribution to independent mobility for children – a qualitative pilot study

Journal of urban mobility, 2023-12, Vol.4, p.100058, Article 100058

2023

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Lifestyle behaviors in Swedish university students before and during the first six months of the COVID-19 pandemic: a cohort study

BMC public health, 2022-06, Vol.22 (1), p.1-1207, Article 1207

2022

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Effects of a three-armed randomised controlled trial using self-monitoring of daily steps with and without counselling in prediabetes and type 2 diabetes-the Sophia Step Study

The international journal of behavioral nutrition and physical activity, 2021-09, Vol.18 (1), p.121-121, Article 121

2021

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Emotion regulation group skills training: a pilot study of an add-on treatment for eating disorders in a clinical setting

Journal of eating disorders, 2020-04, Vol.8 (1), p.12-12, Article 12

2020

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Stronger induction of callose deposition in barley by Russian wheat aphid than bird cherry-oat aphid is not associated with differences in callose synthase or β-1,3-glucanase transcript abundance

Physiologia plantarum, 2009-02, Vol.135 (2), p.150-161

2009

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Criterion validity and test-retest reliability of SED-GIH, a single item question for assessment of daily sitting time

BMC public health, 2019-01, Vol.19 (1), p.17-17, Article 17

2019

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Health care professionals' experiences of supporting persons with metabolic risk factors to increase their physical activity level - a qualitative study in primary care

Scandinavian journal of primary health care, 2023, Vol.41 (2), p.116-131

2023

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Regulatory Standards in Orphan Medicinal Product Designation in the EU

Frontiers in medicine, 2021-06, Vol.8, p.698534-698534

2021

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Differential regulation of 3-aminomethylindole/ N-methyl-3-aminomethylindole N-methyltransferase and gramine in barley by both biotic and abiotic stress conditions

Plant physiology and biochemistry, 2011, Vol.49 (1), p.96-102

2011

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N-Methyltransferase involved in gramine biosynthesis in barley: Cloning and characterization

Phytochemistry (Oxford), 2006-09, Vol.67 (18), p.2002-2008

2006

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Advanced Therapy Medicinal Products for Rare Diseases: State of Play of Incentives Supporting Development in Europe

Frontiers in medicine, 2017-05, Vol.4, p.53-53

2017

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Defining Satisfactory Methods of Treatment in Rare Diseases When Evaluating Significant Benefit–The EU Regulator's Perspective

Frontiers in medicine, 2021-08, Vol.8, p.744625-744625

2021

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Two methylenetetrahydrofolate reductase gene (MTHFR) polymorphisms, schizophrenia and bipolar disorder: An association study

American journal of medical genetics. Part B, Neuropsychiatric genetics, 2008-09, Vol.147B (6), p.976-982

2008

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Distribution of large Emiliania huxleyi in the Central and Northeast Atlantic as a tracer of surface ocean dynamics during the last 25,000years

Marine micropaleontology, 2010-09, Vol.76 (3-4), p.53-66

2010

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Exploring the cessation process from adolescence to young adulthood in individuals with lived experience of nonsuicidal self-injury: a qualitative study

Journal of adolescence (London, England.), 2024-10

2024

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DTNBP1, NRG1, DAOA, DAO and GRM3 Polymorphisms and Schizophrenia: An Association Study

Neuropsychobiology, 2009-01, Vol.59 (3), p.142-150

2009

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Inherited retinal dystrophies and orphan designations in the European Union

European journal of ophthalmology, 2024-03

2024

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Genome-wide profiling of micro RNAS that associate with susceptibility to experimental neuroinflammation

10th Congress of the International-Society-of-Neuroimmunology (ISNI),2010-10-26 - 2010-10-30, 2010, Vol.228 (1-2), p.82

2010

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Fecal Pellets from a Dense Aggregation of Suspension-Feeders in a Stream: An Example of Ecosystem Engineering

Limnology and oceanography, 1998-06, Vol.43 (4), p.719-725

1998

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Therapies for rare diseases: therapeutic modalities, progress and challenges ahead

Emotion regulation group skills training for adolescents and parents: A pilot study of an add-on treatment in a clinical setting

What Is Rare and What Is Orphan? A Guide to the Regulatory Terminology

Recommendations for the design of small population clinical trials

A Randomized Controlled Clinical Trial of Transanal Irrigation Versus Conservative Treatment in Patients With Low Anterior Resection Syndrome After Rectal Cancer Surgery

Predictors associated with an increase in daily steps among people with prediabetes or type 2 diabetes participating in a two-year pedometer intervention

Autonomous shuttles contribution to independent mobility for children – a qualitative pilot study

Lifestyle behaviors in Swedish university students before and during the first six months of the COVID-19 pandemic: a cohort study

Effects of a three-armed randomised controlled trial using self-monitoring of daily steps with and without counselling in prediabetes and type 2 diabetes-the Sophia Step Study

Emotion regulation group skills training: a pilot study of an add-on treatment for eating disorders in a clinical setting

Stronger induction of callose deposition in barley by Russian wheat aphid than bird cherry-oat aphid is not associated with differences in callose synthase or β-1,3-glucanase transcript abundance

Criterion validity and test-retest reliability of SED-GIH, a single item question for assessment of daily sitting time

Health care professionals' experiences of supporting persons with metabolic risk factors to increase their physical activity level - a qualitative study in primary care

Regulatory Standards in Orphan Medicinal Product Designation in the EU

Differential regulation of 3-aminomethylindole/ N-methyl-3-aminomethylindole N-methyltransferase and gramine in barley by both biotic and abiotic stress conditions

N-Methyltransferase involved in gramine biosynthesis in barley: Cloning and characterization

Advanced Therapy Medicinal Products for Rare Diseases: State of Play of Incentives Supporting Development in Europe

Defining Satisfactory Methods of Treatment in Rare Diseases When Evaluating Significant Benefit–The EU Regulator's Perspective

Two methylenetetrahydrofolate reductase gene (MTHFR) polymorphisms, schizophrenia and bipolar disorder: An association study

Distribution of large Emiliania huxleyi in the Central and Northeast Atlantic as a tracer of surface ocean dynamics during the last 25,000years

Exploring the cessation process from adolescence to young adulthood in individuals with lived experience of nonsuicidal self-injury: a qualitative study

DTNBP1, NRG1, DAOA, DAO and GRM3 Polymorphisms and Schizophrenia: An Association Study

Inherited retinal dystrophies and orphan designations in the European Union

Genome-wide profiling of micro RNAS that associate with susceptibility to experimental neuroinflammation

Fecal Pellets from a Dense Aggregation of Suspension-Feeders in a Stream: An Example of Ecosystem Engineering

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