Date du début: 1 janv. 2016,
Date de fin: 31 déc. 2020,
...ial of the safety and efficacy of pre- and/or postnatal MSC transplantation in the severest forms of OI (type III, severe type IV). Transplantation before birth at the onset of disease should lead to greater efficacy and engraftment with less rejection than transplantation after birth. Postnatal transplantation will be evaluated in cases where prenatal diagnosis was not made. The trial’s primary o ...
Date du début: 1 janv. 2016,
Date de fin: 31 déc. 2019,
Severe combined immunodeficiency (SCID) is a devastating rare disorder of immune system development. Affected infants are born without functional immune systems and die within the first year of life unless effective treatment is given. Treatment options are limited to allogeneic haematopoietic stem cell transplantation and autologous stem cell gene therapy. Over the last 15 years, gene therapy for ...
Date du début: 1 nov. 2013,
Date de fin: 31 oct. 2018,
Children are often treated as small adults—not as the special patients they are. Regulations exist to encourage the development of drugs and formulation for children. However, off-patent drugs are often administered to children without appropriate formulations or systematic investigations. Therefore, the paediatric use marketing authorisation (PUMA) has been designed.The EMA Paediatric Working Par ...
Date du début: 1 févr. 2011,
Date de fin: 31 janv. 2017,
Over the last 40 years, treatment for childhood and adolescent cancer has improved greatly; 5- year survival after childhood cancer is now 80% in developed countries. Approximately 1 individual in 750 of young adults is now a childhood cancer survivor. Epidemiologic data on the number of European childhood cancer long-term survivors are not available, but estimates suggest a number between 300,000 ...
Date du début: 1 janv. 2012,
Date de fin: 31 déc. 2016,
Acquired and congenital heart disease can necessitate heart valve replacement. However, current heart valve substitutes are not considered ideal as they need anticoagulation, bearing the risk of bleeding when manufactured from non-organic material, or they degenerate when they derive from animals or human tissue donators (homografts) thereby leading to frequent reoperation especially in the young ...
Date du début: 1 nov. 2012,
Date de fin: 30 avr. 2016,
Duchenne muscular dystrophy (DMD) is a progressive, lethal muscle degenerative condition arising from the absence of dystrophin in skeletal and cardiac muscles. 65% of DMD boys have out-of-frame deletions. Modulation of pre-mRNA splicing by exon skipping is the most promising molecular intervention in DMD. 2 Phase Ib and 2 Phase IIa clinical trials (MDEX Consortium in collaboration with Sarepta Th ...
Date du début: 1 nov. 2010,
Date de fin: 30 avr. 2015,
The emergence of suicidality in patients receiving drug treatment is of concern because of the overall burden and the possible link with completed suicide. The lack of uniform requirements for defining, detecting and recording suicidality and the presence of disease related confounders create major problems. It is possible that Medication-Related Suicidality (MRS) differs from Psychopathology-Rela ...
Date du début: 1 oct. 2009,
Date de fin: 31 mars 2015,
Neonatal hypoxic ischaemic encephalopathy (HIE) occurs in 2-3/1000 live births and is a major cause of both acute mortality and long-term neurodisability. Seizures are the hallmark of HIE. The clinical and electrographic seizure burden in babies with HIE can be considerable and is often not reduced by current antiepileptic drugs. Phenobarbitone remains the first line drug for neonatal seizures des ...
Date du début: 1 mai 2008,
Date de fin: 30 avr. 2012,
"In this proposal, we have mobilized a critical mass of expertise to investigate, on a Europe-wide scale, the natural history and pathophysiology of rare inherited diseases affecting important structures of the kidney. The project will use and develop multiple models (in vitro and in vivo) with the aim to develop preventive, diagnostic and therapeutic interventions that should alleviate the burden ...
Date du début: 15 déc. 2007,
Date de fin: 14 déc. 2011,
The European health care sector is facing future dramatic changes from e.g. the public demand for increased quality of health. Also Europe is facing substantial changes in the energy sector and has set out ambitious targets for abatement of GHG emissions and increased use of renewables. For the health care sector this could result in massive changes in the economy of the sector, which is among the ...
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