TY - JOUR
T1 - Stakeholder cooperation to overcome challenges in orphan medicine development: The example of Duchenne muscular dystrophy
AU - Straub, Volker
AU - Balabanov, Pavel
AU - Bushby, Kate
AU - Ensini, Monica
AU - Goemans, Nathalie
AU - De Luca, Annamaria
AU - Pereda, Alejandra
AU - Hemmings, Robert
AU - Campion, Giles
AU - Kaye, Edward
AU - Arechavala Gomeza, Virginia
AU - Goyenvalle, Aurelie
AU - Niks, Erik
AU - Veldhuizen, Olav
AU - Furlong, Pat
AU - Stoyanova Beninska, Violeta
AU - Wood, Matthew J
AU - Johnson, Alex
AU - Mercuri, Eugenio Maria
AU - Muntoni, Francesco
AU - Sepodes, Bruno
AU - Haas, Manuel
AU - Vroom, Elizabeth
AU - Aartsma Rus, Annemieke
PY - 2016
Y1 - 2016
N2 - Duchenne muscular dystrophy is a rare, progressive, muscle-wasting disease leading to severe disability and premature death. Treatment is currently symptomatic, but several experimental therapies are in development. Implemented care standards, validated outcome measures correlating with clinical benefit, and comprehensive information about the natural history of the disease are essential for regulatory approval of any treatment. However, for Duchenne muscular dystrophy and other rare diseases, these requirements are not always in place when potential therapies enter the clinical trial phase. A cooperative effort of stakeholders in Duchenne muscular dystrophy-including representatives from patients' groups, academia, industry, and regulatory agencies-is aimed at addressing this shortfall by identifying strategies to overcome challenges, developing the tools needed, and collecting relevant data. An open and constructive dialogue among European stakeholders has positively affected development of treatments for Duchenne muscular dystrophy; this approach could serve as a paradigm for development of treatments for rare diseases in general.
AB - Duchenne muscular dystrophy is a rare, progressive, muscle-wasting disease leading to severe disability and premature death. Treatment is currently symptomatic, but several experimental therapies are in development. Implemented care standards, validated outcome measures correlating with clinical benefit, and comprehensive information about the natural history of the disease are essential for regulatory approval of any treatment. However, for Duchenne muscular dystrophy and other rare diseases, these requirements are not always in place when potential therapies enter the clinical trial phase. A cooperative effort of stakeholders in Duchenne muscular dystrophy-including representatives from patients' groups, academia, industry, and regulatory agencies-is aimed at addressing this shortfall by identifying strategies to overcome challenges, developing the tools needed, and collecting relevant data. An open and constructive dialogue among European stakeholders has positively affected development of treatments for Duchenne muscular dystrophy; this approach could serve as a paradigm for development of treatments for rare diseases in general.
KW - Neurology (clinical)
KW - Neurology (clinical)
UR - https://publicatt.unicatt.it/handle/10807/93156
UR - https://www.scopus.com/inward/citedby.uri?partnerID=HzOxMe3b&scp=84973316149&origin=inward
UR - https://www.scopus.com/inward/record.uri?partnerID=HzOxMe3b&scp=84973316149&origin=inward
U2 - 10.1016/S1474-4422(16)30035-7
DO - 10.1016/S1474-4422(16)30035-7
M3 - Article
SN - 1474-4422
VL - 15
SP - 882
EP - 890
JO - LANCET NEUROLOGY
JF - LANCET NEUROLOGY
IS - 8
ER -