Emerging drugs for the treatment of mucopolysaccharidoses

Expert Opinion on Emerging Drugs

Volumn 21, Issue 1, 2016, Pages 9-26

  Giugliani, Roberto ^a,b   Federhen, Andressa ^a,b   Vairo, Filippo ^a   Vanzella, Cláudia ^a,b   Pasqualim, Gabriela ^a,b   Da Silva, Letícia Machado Rosa ^a   Giugliani, Luciana ^a   De Boer, Ana Paula Kurz ^a   De Souza, Carolina Fishinger Moura ^a   Matte, Ursula ^a,b   Baldo, Guilherme ^a,b  

^a HOSPITAL DE CLÍNICAS DE PORTO ALEGRE   (Brazil)

^b FEDERAL UNIVERSITY OF RIO GRANDE DO SUL   (Brazil)

Author keywords

emerging treatments; enzyme replacement therapy; gene therapy; glycosaminoglycans; lysosomal storage diseases; Mucopolysaccharidoses

Indexed keywords

HYBRID PROTEIN; NANOPARTICLE; PENTOSAN POLYSULFATE; RHODAMINE B;

BLOOD BRAIN BARRIER; BONE MARROW TRANSPLANTATION; ENZYME REPLACEMENT; ENZYME THERAPY; GENE THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HUNTER SYNDROME; HURLER SYNDROME; MARKET; MICROENCAPSULATION; MORQUIO SYNDROME; MUCOPOLYSACCHARIDOSIS; NANOTECHNOLOGY; NONHUMAN; REVIEW; SANFILIPPO SYNDROME; STOP CODON; SUBSTRATE REDUCTION THERAPY; ANIMAL; COGNITION DISORDERS; COMPLICATION; DISEASE COURSE; DRUG DESIGN; MUCOPOLYSACCHARIDOSES; PATHOPHYSIOLOGY; PROCEDURES;

ANIMALS; COGNITION DISORDERS; DISEASE PROGRESSION; DRUG DESIGN; ENZYME REPLACEMENT THERAPY; GENETIC THERAPY; HUMANS; MUCOPOLYSACCHARIDOSES;

EID: 84959544287     PISSN: 14728214     EISSN: 17447623     Source Type: Journal    
DOI: 10.1517/14728214.2016.1123690     Document Type: Review

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