The RUDY study platform – a novel approach to patient driven research in rare musculoskeletal diseases

[1] Schieppati A(2008)Why rare diseases are an important medical and social issue Lancet 371 2039-41

[2] Henter JI(2009)Clinical research for rare disease: opportunities, challenges, and solutions Mol Genet Metab 96 20-6

[3] Daina E(2006)A journey of hope: lessons learned from studies on rare diseases and orphan drugs J Intern Med 260 1-10

[4] Aperia A(2013)Clinical trials in rare disease: challenges and opportunities J Child Neurol 28 1142-50

[5] Griggs RC(2014)Toward a functional definition of a "rare disease" for regulatory authorities and funding agencies Value Health 17 757-61

[6] Batshaw M(2015)Dynamic consent: a patient interface for twenty-first century research networks Eur J Hum Genet 23 141-6

[7] Dunkle M(2014)The EPIRARE proposal of a set of indicators and common data elements for the European platform for rare disease registration Arch Public Health 72 35-43

[8] Gopal-Srivastava R(2001)EQ-5D: a measure of health status from the EuroQol Group Ann Med 33 337-40

[9] Kaye E(1993)Short form 36 (SF36) health survey questionnaire: normative data for adults of working age BMJ 306 1437-3

[10] Krischer J(1992)The Extended Activities of Daily Living scale: a further validation Disabil Rehabil 14 41-213

[1] Schieppati A(2008)Why rare diseases are an important medical and social issue Lancet 371 2039-41

[2] Henter JI(2009)Clinical research for rare disease: opportunities, challenges, and solutions Mol Genet Metab 96 20-6

[3] Daina E(2006)A journey of hope: lessons learned from studies on rare diseases and orphan drugs J Intern Med 260 1-10

[4] Aperia A(2013)Clinical trials in rare disease: challenges and opportunities J Child Neurol 28 1142-50

[5] Griggs RC(2014)Toward a functional definition of a "rare disease" for regulatory authorities and funding agencies Value Health 17 757-61

[6] Batshaw M(2015)Dynamic consent: a patient interface for twenty-first century research networks Eur J Hum Genet 23 141-6

[7] Dunkle M(2014)The EPIRARE proposal of a set of indicators and common data elements for the European platform for rare disease registration Arch Public Health 72 35-43

[8] Gopal-Srivastava R(2001)EQ-5D: a measure of health status from the EuroQol Group Ann Med 33 337-40

[9] Kaye E(1993)Short form 36 (SF36) health survey questionnaire: normative data for adults of working age BMJ 306 1437-3

[10] Krischer J(1992)The Extended Activities of Daily Living scale: a further validation Disabil Rehabil 14 41-213