Multifaceted roles of ultra-rare and rare disease patients/parents in drug discovery

Drug Discovery Today

Volumn 18, Issue 21-22, 2013, Pages 1043-1051

  Wood, Jill ^a,b   Sames, Lori ^c,d   Moore, Allison ^e   Ekins, Sean ^{b,d,f,g,h,i,j}  

^a Jonah's Just Begun   (United States)

^b Phoenix Nest   (United States)

^c Hannah's Hope Fund   (United States)

^d BioGAN Therapeutics   (United States)

^e Hereditary Neuropathy Foundation   (United States)

^f Collaborations in Chemistry ^*  (United States)

^g Collaborative Drug Discovery ^*  (United States)

^h UNIVERSITY OF MARYLAND   (United States)

ⁱ RUTGERS ROBERT WOOD JOHNSON MEDICAL SCHOOL   (United States)

^j University of North Carolina   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

AGALSIDASE BETA; COMPLEMENT COMPONENT C1S INHIBITOR; ECULIZUMAB; GALSULFASE; IDURONATE 2 SULFATASE; IMIGLUCERASE; IVACAFTOR; PERIPHERAL MYELIN PROTEIN 22; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; VELAGLUCERASE ALFA;

ALZHEIMER DISEASE; BONE MARROW TRANSPLANTATION; DIFFERENTIAL DIAGNOSIS; DISEASE COURSE; DRUG COST; DRUG DEVELOPMENT; DRUG INDUSTRY; DUCHENNE MUSCULAR DYSTROPHY; EATON LAMBERT SYNDROME; ENZYME ACTIVITY; FABRY DISEASE; FAMILY; FRIEDREICH ATAXIA; GAUCHER DISEASE; GENE DUPLICATION; GIANT AXONAL NEUROPATHY; GLYCOGEN STORAGE DISEASE TYPE 2; HUMAN; INTERMEDIATE FILAMENT; LYSOSOME STORAGE DISEASE; MULTIPLE SCLEROSIS; MUSCULAR DYSTROPHY; MYELINATION; NERVE CONDUCTION; NERVE FIBER DEGENERATION; NERVE FIBER TRANSPORT; NIEMANN PICK DISEASE; NONHUMAN; PARENT; PARKINSON DISEASE; PATENT; PATIENT; PHENOTYPE; QUALITY OF LIFE; RARE DISEASE; REVIEW; SANFILIPPO SYNDROME; SENSORY NEUROPATHY;

CHILD; COOPERATIVE BEHAVIOR; DRUG DISCOVERY; DRUG INDUSTRY; HUMANS; MODELS, ORGANIZATIONAL; ORPHAN DRUG PRODUCTION; PARENTS; PATIENT ADVOCACY; RARE DISEASES; RESEARCH;

EID: 84886953083     PISSN: 13596446     EISSN: 18785832     Source Type: Journal    
DOI: 10.1016/j.drudis.2013.08.006     Document Type: Short Survey

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