x

Search for a research project

* (*) mandatory field

Other search option(s)

38 Result(s)

Funded by an IRDiRC member =

Research projects

CANADA

Ontario
OTTAWA

Funded by an IRDiRC memberEmerging team in rare diseases: achieving the "triple aim" for inborn errors of metabolism
Children's Hospital of Eastern Ontario
Newborn Screening Ontario

CANADA

Ontario
OTTAWA, ONTARIO

Funded by an IRDiRC memberEmerging team in rare diseases: achieving the "triple aim" for inborn errors of metabolism
University of Ottawa
Department of Epidemiology and Community Medicine

CANADA

Québec
MONTRÉAL

Clinical, radiological and genetic characterization of new forms of leukodystrophies
Montreal Children's Hospital Research Institute - McGill University
Pediatric Neurodegenerative Laboratory

FRANCE

HAUTS-DE-FRANCE
LILLE

ID2STOP Orphan : InDividualized Drug Selection Technology for Orphan Patients
Faculté des Sciences Pharmaceutiques et Biologiques de Lille
APTEEUS: le patient au coeur de la découverte de son traitement

FRANCE

ILE-DE-FRANCE
PARIS

Funded by an IRDiRC memberLEUKOTREAT: ethical impacts of therapeutic challenges in leukodystrophies (WP5) - FR
Centre Universitaire des Saints-Pères - Faculté de Médecine
Laboratoire d'éthique médicale et médecine légale

GERMANY

Mecklenburg-Vorpommern
ROSTOCK

Biomarker for Krabbe Disease (BioKrabbe): An International, multicentre, epidemiological protocol
Albrecht-Kossel-Institute for Neuroregeneration (AKos)
Albrecht-Kossel-Institut für Neurodegeneration

GERMANY

Niedersachsen
GÖTTINGEN

Clinical and molecular characterization of genetically determined unclear white matter disorders
Universitätsmedizin Göttingen
Klinik für Kinder- und Jugendmedizin

ITALY

CAMPANIA
NAPOLI

Funded by an IRDiRC memberTelethon Undiagnosed Disease Program - Revised Proposal
Azienda Ospedaliera Universitaria - Seconda Università degli Studi di Napoli
Laboratorio di Genetica Medica

ITALY

LIGURIA
GENOVA

New technologies and new strategies for neonatal screening
IRCCS Istituto G. Gaslini - Ospedale Pediatrico
Laboratorio per lo Studio degli Errori Congeniti del Metabolismo

ITALY

LOMBARDIA
MILANO

Funded by an IRDiRC memberVector safety studies
IRCCS Ospedale San Raffaele
Centro di Genomica, Bioinformatica e Biostatistica - Unità di Genetica Umana e Molecolare

ITALY

LOMBARDIA
MILANO

Combined approaches based on gene transfer and neural stem cells (NSC) for Metachromatic leukodystrophy and Globoid cell leukodystrophy
Istituto San Raffaele Telethon per la Terapia Genica - TIGET
Istituto San Raffaele Telethon per la Terapia Genica

ITALY

LOMBARDIA
MILANO

Funded by an IRDiRC memberHsc-based ex vivo gene therapy of metachromatic and globoid leukodystrophy
Istituto San Raffaele Telethon per la Terapia Genica - TIGET
Unità di Ricerca Clinica Pediatrica

ITALY

LOMBARDIA
MILANO

Funded by an IRDiRC memberLEUKOTREAT: innovative gene and cell therapies in leukodystrophies (WP4) - IT
Istituto San Raffaele Telethon per la Terapia Genica - TIGET
Unità di Ricerca Clinica Pediatrica

ITALY

LOMBARDIA
MILANO

Funded by an IRDiRC memberCellule staminali ematopoietiche per la terapia genica nel trattamento della leucodistrofia globoide
Istituto San Raffaele Telethon per la Terapia Genica - TIGET
Unità di Ricerca Clinica Pediatrica

PORTUGAL

NORTE
PORTO

Splicing therapeutics for patients affected by lisosomal storage disorders.
Instituto Nacional de Saúde Dr. Ricardo Jorge - Porto
Unidade de Investigação & Desenvolvimento; Departamento de Genética

PORTUGAL

NORTE
PORTO

The crosstalk between lipid antigen presentation and the pathogenic mechanisms of Lysosomal Storage Diseases
Instituto de Biologia Molecular e Celular
Unidade de Biologia do Lisossoma e do Peroxissoma

SWEDEN

Stockholms läns landsting
STOCKHOLM

Inborn errors of endocrinology and metabolism
Karolinska Institutet - Solna
Department of Molecular Medicine and Surgery

UNITED KINGDOM

Cambridgeshire
CAMBRIDGE

Funded by an IRDiRC memberActive BRIDGE Studies - Specialist Pathology: Evaluating Exomes in Diagnostics (SPEED)
Wellcome Trust/MRC Building
Cambridge Institute for Medical Research

UNITED KINGDOM

Lothian
EDINBURGH

Genetic disorders of human brain development
Western General Hospital
MRC Human Genetics Unit

UNITED KINGDOM

Oxfordshire
BEGBROKE

Multicentric Research projects