Key challenges in the treatment of rare pediatric skeletal genetic disorders: from bench to bedside.
Genentech Safety Assessment, 1 DNA Way, South San Francisco, CA 94080, USA. Electronic address: lorget.florence@gene.com.
Florence
F
Lorget
Imagine Institute, INSERM U1163, Paris Descartes University, 24 Boulevard du Montparnasse, 75015 Paris, France. Electronic address: laurence.legeai-mallet@inserm.fr.
Laurence
L
Legeai-Mallet
D000367:Age Factors; D000818:Animals; D001847:Bone Diseases / Q000175:diagnosis / Q000235:genetics / Q000628:therapy*; D002648:Child; D030342:Genetic Diseases, Inborn / Q000175:diagnosis / Q000235:genetics / Q000628:therapy*; D005819:Genetic Markers; D020022:Genetic Predisposition to Disease; D006801:Humans; D010641:Phenotype; D035583:Rare Diseases / Q000175:diagnosis / Q000235:genetics / Q000628:therapy*; D057170:Translational Research, Biomedical / Q000379:methods*; D016896:Treatment Outcome
D005819:Genetic Markers