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7 X 11.5 Long Title.P65 Cambridge University Press 978-0-521-51781-2 - Small Molecule Therapy for Genetic Disease Edited by Jess G. Thoene Index More information Index Abderhalden, Emil, 101 long-term therapy results, 147–149 absolute oral bioavailability, 37 side effects, 147 acquired copper deficiency, 202–203 antagonists, 46 active tubular secretion, 43 apomorphine, 12 acute hereditary tyrosinemia, 115 apoptosis, in cysteamine treatment, for adenosine triphosphate (ATP) nephropathic cystinosis, 103 ATP7A, 203 apparent first-order kinetics, 40–41 distal hereditary motor neuropathy, apparent volume of distribution, 39 206–207 argininosuccinate lyase (AL) deficiency, Menkes disease, 203 135 OHS, 206 argininosuccinate synthetase (AS) nephropathic cystinosis, 103 deficiency, 135 agonists, 46 AS deficiency. See argininosuccinate AKU. See alkaptonuria synthetase deficiency AL deficiency. See argininosuccinate lyase ATP. See adenosine triphosphate deficiency ATP7A, 203 ALA. See aminolevulinic acid distal hereditary motor neuropathy, alkaptonuria (AKU), 114, 118–121 206–207 cardiac involvement, 119–120 Menkes disease, 203 clinical presentation, 119 OHS, 206 arthritis as, 119 diagnosis, 121 basal ganglion diseases, 60 dietary therapies, 120 biotin therapy, 66 genetic factors, 121 inheritance factors, 63 HGA, 118–119 Bench-To-Bedside Research Program, HPPD inhibition, 124 25 incidence rates, 118–119 betaine, in homocystinuria treatment, nitisinone therapy, 125 176–179 future developments, 130 clinical evidence of, 176–177 side effects, 127 development, 176–177 tyrosine levels, 129–130 dosage, 177–178 pathophysiology, 121 FDA status, 179 renal transplants, 120 future developments, 179 vitamin therapy, 120 for remethylation disorders, 177 Alpha-1 Foundation, 21 cblC disorder, 177 ambrisentan, 11 MTHFR, 177 amino acylation products, 142–144 side effects, 178–179 aminolevulinic acid (ALA), 117–118 case studies, 178–179 Ammonul, 144–145 Bickel, Horst, 76 alternative therapy uses, 149 bioavailability, of drugs, 37–38 dosage, 146 absolute oral, 37 overdosage, 147 bioequivalence and, 38 FDA status, 147 relative, 37 HIP, 144–145 studies, 37 213 © in this web service Cambridge University Press www.cambridge.org Cambridge University Press 978-0-521-51781-2 - Small Molecule Therapy for Genetic Disease Edited by Jess G. Thoene Index More information 214 Index bioequivalence, 38 primary, 88–92 FDA guidelines for, 38 case reports, 89–90 biomarkers, in drug development, 49–50 definition, 88 Biopten. See sapropterin dihydrochloride incidence rate, 88–89 biotin, 64–66 L-carnitine therapy for, 90–92 biosynthetic pathways, 64 in muscles, 89 cGMP activity enhancement and, 62 from OCTN2 deficiency, 89–90 dosing, 65 secondary, 88, 92–96 FDA recommendations, 65–66 clinical presentation, 92–94 gene transcription regulation, 61–62 clinical study history, 95 metabolism of, responsive disorders and, L-carnitine therapy for, 94–96 58–59 pathophysiology, 92 preparation for, 64–65 screening, 95 side effects, 65 symptoms, 92 structure, 64 Carnitor, 94 UV light and, 64 cblC disorder, 175 biotin therapy, 66 betaine therapy, 177 basal ganglion diseases, 66 CCO. See cytochrome c oxidase biotinidase deficiency, 66 CDME. See cystine dimethyl ester HCS deficiency, 66 CETT. See Collaboration, Education and biotinidase deficiency, 60 Test Translation program biotin therapy, 66 cGMP. See cyclic guanosine inheritance factors, 63 monophosphate biotin-responsive disorders, 57–64 chemical chaperones, 156, 168 basal ganglion diseases, 60 Fabry disease, 168 inheritance factors, 63 Gaucher disease, 167–168 biotin metabolism, 58–59 children. See also neonates, Menkes disease biotin therapy, 66 and biotinidase deficiency, 60 biotin-responsive disorders, 60–61 inheritance factors, 63 pharmacokinetic variables in, 51–52 in children, 60–61 Wilson disease in, zinc therapy for, 195 clinical presentation, 59–60 chitotriosidase, 165 COA deficiency, 57 chronic hereditary tyrosinemia, 115–116 HCS deficiency, 57 chronic hyperammonemia, 138–139 clinical presentation, 59 clearance, of drugs, 39–40 inheritance factors, 62–63 elimination, 39–40 hearing loss from, 61 hepatic, mechanisms and determinants immunological abnormalities, 61 of, 40 inheritance factors, 62–64 blood flow and, 40 MCD, 57 protein binding and, 40 pathophysiology, 60–62 intercompartmental, 39 in brain tissue, 61 renal, mechanisms and determinants of, causative factors, 60 40 PCC deficiency, 57 Fick principle, 40 screening programs, 62 GFR and, 40 symptoms, 59 Clinical and Translational Science Award bone marrow transplantation, for (CTSA), 25 lysosomal storage disorders, 156 COA. See ␤-methylcrotonyl-coenzyme A bosentan, 11 CoA. See coenzyme A Buphenyl. See sodium phenylbutyrate cobalamin, 72–73 coenzymes, 69 carbamoyl phosphate synthetase (CPS) deficiency, 72 deficiency, 135 mechanism of action, 73 cardiomyopathy, HT and, 129 metabolism, 69 carnitine deficiency disorders as MMA treatment therapy, 70, 73 L-carnitine therapy, 86–87 clinical effects, 73 mortality rates, 94–95 dosage, 73 for primary carnitine deficiency, 90–92 future developments, 74 for secondary carnitine deficiency, natural forms, 69 94–96 pharmaceutical formulations, 72–73 © in this web service Cambridge University Press www.cambridge.org Cambridge University Press 978-0-521-51781-2 - Small Molecule Therapy for Genetic Disease Edited by Jess G. Thoene Index More information Index 215 processing defects, 71–72 mutation inheritance, 104 Cb1A, 71 renal failure from, 109 Cb1B, 71 results, 109–110 Cb1C, 71–72 side effects, 108 Cb1D, 72 Stevens-Johnson syndrome from, 108 Cb1F, 72 vomiting, 108 Cockroft-Gault equation, 50 Cystic Fibrosis Foundation, 20–21 coenzyme A (CoA), 86–87 cystine dimethyl ester (CDME), 103 Collaboration, Education and Test cystinosis. See nephropathic cystinosis Translation (CETT) program, 22–23 cystinuria, 101 genetic mutation information collection, cytochrome c oxidase (CCO), 205 23 goals, 22 distal hereditary motor neuropathy, copper, as trace element, 202 206–207 copper chloride, 208 distribution, of drugs, 38–39 copper deficiency syndromes, 202–211 VD, 38–39 acquired, 202–203 apparent, 39 distal hereditary motor neuropathy, drug absorption, 37 206–207 drug development, 47–50. See also inherited, 203–207 pharmacodynamics; Menkes disease, 202–205 pharmacokinetics animal models, 207 biomarkers, 49–50 ATP7A, 203 dose-escalation schemes, 48–49 brain pathology, 205 Fibonacci, 48 CCO activity, 205 PGDE, 48–49 clinical features, 204–205 exploratory trials, with clinical presentation, 203–204 nonpharmacological doses, 49 diagnosis, 204 FIH dosing estimates, 48 during neonatal period, 205 drug metabolism, 40–41 PAM deficiency, 204–205 clinical approaches to, 51 phenotypes, 204 enzymes for, 42 X-linked, 203 CYP, 41–42 OHS, 206 kinetics, 40–41 molecular basis, 206 apparent first-order, 40–41 small copper complexes, in treatment saturable of Michaelis-Menten type, therapies, 207–209 41 transport mechanisms to central nervous reactions, 41 system, 209–210 hepatic cells, 41 copper gluconate, 207–209 Phase I, 41 copper histidine, 208 transport, 41–42 copper sulfate, 208 CYP enzymes, 41–42 CPS deficiency. See carbamoyl phosphate genomics, 41–42 synthetase deficiency drug-dosing, pharmacokinetic principles, CTSA. See Clinical and Translational 43–45 Science Award continuous infusion, 44 cyclic guanosine monophosphate (cGMP), design criteria, 45 62 multiple regimens, 44–45 CYP enzymes, 41–42 plateau principle, 43–44 racial distribution, 42 single regimens, 44 Cystagon. See cysteamine drug-receptor interactions, 46 cysteamine, 104–106 agonists, 46 cystine storage, 105 antagonists, 46 hygroscopic nature, 104 dose-response relationship, 46–47 structure, 104 drug efficacy, 46 as treatment, for nephropathic cystinosis, 101–106, 111 elimination clearance, 39–40 apoptosis, 103 EMEA. See European Medicines Agency dosage, 107–108 enzyme replacement therapy, for lysosomal FDA status, 109 storage disorders, 155–156, 168 future applications, 110–111 epoprostenol, 12 © in this web service Cambridge University Press www.cambridge.org Cambridge University Press 978-0-521-51781-2 - Small Molecule Therapy for Genetic Disease Edited by Jess G. Thoene Index More information 216 Index European Medicines Agency (EMEA), 6 for neglected diseases, 30 OTT, 30 FAA. See fumarylacetoacetate Gallin, John, 24 Fabry disease, 155 GARD. See Gaining Access to Research chemical chaperones, 168 Discoveries combination therapy for, 168 Gaucher disease, 155 enzyme replacement therapy, 155–156, alternative small molecular entities, 168 165–168 synthesis inhibition therapy, 156–157 chemical chaperones, 167–168 Fanconi syndrome, 101, 109 miglustat, 165–167 MAA as factor, 117 chitotriosidase as biomarker, 165 Faucett, Andrew, 23 combination therapy for, 168 FDA. See Food and Drug Administration enzyme replacement therapy, 155–156, FDAAA. See Food and Drug Administration 168 Amendments Act Genz-112638 in treatment therapy, 166 FDCA. See Federal Food, Drug, and miglustat in treatment therapy, 166 Cosmetics Act Phase II studies, 164 Federal Food, Drug, and Cosmetics Act synthesis inhibition therapy, 156–157 (FDCA) (U.S.), 3 gene therapy, for lysosomal storage Fibonacci scheme, 48 disorders, 156, 168 Fick principle, 40 genetic disorders, orphan drugs for FIH dose. See first-in-human dose clinical development of, 14 first-in-human (FIH) dose, 48 nonbiological, FDA approval of, 9, 12–15 Folling, Asbjorn, 76 Genz-112638, 163–165 Food and Drug Administration (FDA) Gaucher disease therapy, 166 Ammonul status, 147 miglustat v., as treatment therapy, 167
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