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2. Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy.

Author

Wong, Brenda, Flanigan, Kevin, Wilson, Rosamund, de Kimpe, Sjef, Lourbakos, Afrodite, Lin, Zhengning, Campion, Giles, and McDonald, Craig

Abstract

OBJECTIVE: This double-blind, randomized, placebo-controlled Phase 2 study (NCT01462292) assessed the 24-week efficacy, safety, tolerability, and pharmacokinetics of two different subcutaneous drisapersen doses, and the 24-week off-dose persistent effect, in ambulant Duchenne muscular dystrophy (DMD) patients. METHODS: Male DMD patients (≥5 years; time to rise from floor ≤15 s) were randomized to drisapersen 3 mg/kg/week, 6 mg/kg/week or placebo. The primary efficacy endpoint was change from baseline in 6-minute walking distance (6MWD) at week 24. Secondary endpoints included changes in timed function tests, muscle strength, and pulmonary function tests. RESULTS: Fifty-one patients were randomized to placebo (N = 16), drisapersen 3 mg/kg/week (N = 17) or 6 mg/kg/week (N = 18). All but 2 patients had baseline rise from floor time

Published
2018

5. A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy

Author

Araujo, A., Bertini, E., Born, P., Cances, C., Chabrol, B., Chae, J.-H., Colomer Oferil, J., Comi, G.P., Cuisset, J.-M., D'Anjou, G., Desguerre, I., Erazo Torricelli, R., Escobar, R., Feder, D., Ferlini, A., Giugliani, R., Henricson, E., Herczegfalvi, A., Jong, Y.-J., Kimura, S., Kirschner, J.-B., Kleinsteuber, K., Kostera-Pruszczyk, A., Kudr, M., Mueller-Felber, W., Niks, E.H., Ogata, K., Palermo, C., Pane, M., Pascual, I., Pereon, Y., Raskin, S., Rasmussen, M., Reed, U., Schara, U., Selby, K., Sobreira, C., Takeshima, Y., Vilchez Padilla, J.J., Vita, G., Vondracek, P., Wiegand, G., Wilichowski, E., Goemans, Nathalie, Mercuri, Eugenio, Belousova, Elena, Komaki, Hirofumi, Dubrovsky, Alberto, McDonald, Craig M., Kraus, John E., Lourbakos, Afrodite, Lin, Zhengning, Campion, Giles, Wang, Susanne X., and Campbell, Craig

Published
2018
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6. SLN124, a GalNAc conjugated 19‐mer siRNA targeting tmprss6, reduces plasma iron and increases hepcidin levels of healthy volunteers

Author

Porter, John B., primary, Scrimgeour, Alison, additional, Martinez, Alberto, additional, James, Leo, additional, Aleku, Manuela, additional, Wilson, Rosamund, additional, Muckenthaler, Martina, additional, Boyce, Malcolm, additional, Wilkes, Denise, additional, Schaeper, Ute, additional, and Campion, Giles V., additional

Published
2023
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9. Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy

Author

Straub, Volker, Balabanov, Pavel, Bushby, Kate, Ensini, Monica, Goemans, Nathalie, De Luca, Annamaria, Pereda, Alejandra, Hemmings, Robert, Campion, Giles, Kaye, Edward, Arechavala-Gomeza, Virginia, Goyenvalle, Aurelie, Niks, Erik, Veldhuizen, Olav, Furlong, Pat, Stoyanova-Beninska, Violeta, Wood, Matthew J, Johnson, Alex, Mercuri, Eugenio, Muntoni, Francesco, Sepodes, Bruno, Haas, Manuel, Vroom, Elizabeth, and Aartsma-Rus, Annemieke

Published
2016
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10. Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study

Author

Voit, Thomas, Topaloglu, Haluk, Straub, Volker, Muntoni, Francesco, Deconinck, Nicolas, Campion, Giles, De Kimpe, Sjef J, Eagle, Michelle, Guglieri, Michela, Hood, Steve, Liefaard, Lia, Lourbakos, Afrodite, Morgan, Allison, Nakielny, Joanna, Quarcoo, Naashika, Ricotti, Valeria, Rolfe, Katie, Servais, Laurent, Wardell, Claire, Wilson, Rosamund, Wright, Padraig, and Kraus, John E

Published
2014
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14. Single Ascending Dose Study of a Short Interfering RNA Targeting Lipoprotein(a) Production in Individuals With Elevated Plasma Lipoprotein(a) Levels

Author

Nissen, Steven E., primary, Wolski, Kathy, additional, Balog, Craig, additional, Swerdlow, Daniel I., additional, Scrimgeour, Alison C., additional, Rambaran, Curtis, additional, Wilson, Rosamund J., additional, Boyce, Malcom, additional, Ray, Kausik K., additional, Cho, Leslie, additional, Watts, Gerald F., additional, Koren, Michael, additional, Turner, Traci, additional, Stroes, Erik S., additional, Melgaard, Carrie, additional, and Campion, Giles V., additional

Published
2022
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19. Treatment and prevention of lipoprotein(a)-mediated cardiovascular disease: the emerging potential of RNA interference therapeutics.

Author

Swerdlow, Daniel I, Rider, David A, Yavari, Arash, Lindholm, Marie Wikström, Campion, Giles V, and Nissen, Steven E

Subjects
CARDIOVASCULAR diseases, SMALL interfering RNA, AORTIC valve diseases, MAJOR adverse cardiovascular events, CORONARY disease, PERIPHERAL vascular diseases, GENE targeting, CHOLESTERYL ester transfer protein
Abstract

Lipid- and lipoprotein-modifying therapies have expanded substantially in the last 25 years, resulting in reduction in the incidence of major adverse cardiovascular events. However, no specific lipoprotein(a) [Lp(a)]-targeting therapy has yet been shown to reduce cardiovascular disease risk. Many epidemiological and genetic studies have demonstrated that Lp(a) is an important genetically determined causal risk factor for coronary heart disease, aortic valve disease, stroke, heart failure, and peripheral vascular disease. Accordingly, the need for specific Lp(a)-lowering therapy has become a major public health priority. Approximately 20% of the global population (1.4 billion people) have elevated levels of Lp(a) associated with higher cardiovascular risk, though the threshold for determining 'high risk' is debated. Traditional lifestyle approaches to cardiovascular risk reduction are ineffective at lowering Lp(a). To address a lifelong risk factor unmodifiable by non-pharmacological means, Lp(a)-lowering therapy needs to be safe, highly effective, and tolerable for a patient population who will likely require several decades of treatment. N-acetylgalactosamine-conjugated gene silencing therapeutics, such as small interfering RNA (siRNA) and antisense oligonucleotide targeting LPA , are ideally suited for this application, offering a highly tissue- and target transcript-specific approach with the potential for safe and durable Lp(a) lowering with as few as three or four doses per year. In this review, we evaluate the causal role of Lp(a) across the cardiovascular disease spectrum, examine the role of established lipid-modifying therapies in lowering Lp(a), and focus on the anticipated role for siRNA therapeutics in treating and preventing Lp(a)-related disease. [ABSTRACT FROM AUTHOR]

Published
2022
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20. Systemic Administration of PRO051 in Duchenneʼs Muscular Dystrophy

Author

Goemans, Nathalie M., Tulinius, Mar, van den Akker, Johanna T., Burm, Brigitte E., Ekhart, Peter F., Heuvelmans, Niki, Holling, Tjadine, Janson, Anneke A., Platenburg, Gerard J., Sipkens, Jessica A., Ad Sitsen, J. M., Aartsma-Rus, Annemieke, van Ommen, Gert-Jan B., Buyse, Gunnar, Darin, Niklas, Verschuuren, Jan J., Campion, Giles V., de Kimpe, Sjef J., and van Deutekom, Judith C.

Published
2011
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22. Levels of keratan sulfate in the serum and synovial fluid of patients with osteoarthritis of the knee

Author

Campion, Giles V., McCrae, Fiona, Schnitzer, Thomas J., Lenz, Mary Ellen, Dieppe, Paul A., and Thonar, Eugene J.-M.

Subjects
Proteoglycans -- Measurement, Osteoarthritis -- Development and progression, Health
Abstract

In joints, cartilage tissue is composed of chondrocytes (cartilage cells) embedded in a support matrix containing collagens (the body's major structural protein) and proteoglycans (PGs), which are composed of various proportions of proteins and sugar polymers such as keratan sulfate (KS). PGs are highly hydrated, which can greatly increase the volume occupied by PGs, but the extent of swelling is limited by the collagens. This arrangement allows shape changes and reversible deformation of cartilage. Cartilage breakdown products such as KS can be measured in the blood and provide information about cartilage degradation. Cartilage PG degradation is accelerated in osteoarthritis (OA), with PG depletion leading to joint tissue degeneration. Monitoring disease activity and progression is important for treatment, yet is currently inadequate. To determine if measurement of KS levels would be useful in evaluating the course of OA, KS levels in blood and synovial (joint) fluid were evaluated in 125 patients (90 female) with OA of the knee. Blood KS levels in the OA patients were higher than in patients without OA. In addition, levels of KS were significantly higher in men than in women, despite the weak correlation between KS levels and height and weight. Blood KS levels were significantly but weakly related to disease involvement of multiple joints in women, as indicated by the presence of Heberden's nodes (nodules on outermost finger joints) and hip symptoms. Blood KS levels did not correlate with disease measures such as severity, disease duration, medications used, occurrence of disease flares, or involvement of other joint sites. Regardless of the absolute blood KS levels in individuals, the levels tended to be stable over time. KS levels were significantly higher in synovial fluid than blood, and these did not correlate. Synovial levels of KS correlated negatively, or opposite to, joint narrowing as seen on X-ray. The report suggests that blood KS levels would not be useful for diagnosis, but might be helpful in monitoring patients' responses to therapy. (Consumer Summary produced by Reliance Medical Information, Inc.)

Published
1991

24. A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy

Author

Goemans, Nathalie, primary, Mercuri, Eugenio, additional, Belousova, Elena, additional, Komaki, Hirofumi, additional, Dubrovsky, Alberto, additional, McDonald, Craig M., additional, Kraus, John E., additional, Lourbakos, Afrodite, additional, Lin, Zhengning, additional, Campion, Giles, additional, Wang, Susanne X., additional, Campbell, Craig, additional, Araujo, A., additional, Bertini, E., additional, Born, P., additional, Cances, C., additional, Chabrol, B., additional, Chae, J.-H., additional, Colomer Oferil, J., additional, Comi, G.P., additional, Cuisset, J.-M., additional, D'Anjou, G., additional, Desguerre, I., additional, Erazo Torricelli, R., additional, Escobar, R., additional, Feder, D., additional, Ferlini, A., additional, Giugliani, R., additional, Henricson, E., additional, Herczegfalvi, A., additional, Jong, Y.-J., additional, Kimura, S., additional, Kirschner, J.-B., additional, Kleinsteuber, K., additional, Kostera-Pruszczyk, A., additional, Kudr, M., additional, Mueller-Felber, W., additional, Niks, E.H., additional, Ogata, K., additional, Palermo, C., additional, Pane, M., additional, Pascual, I., additional, Pereon, Y., additional, Raskin, S., additional, Rasmussen, M., additional, Reed, U., additional, Schara, U., additional, Selby, K., additional, Sobreira, C., additional, Takeshima, Y., additional, Vilchez Padilla, J.J., additional, Vita, G., additional, Vondracek, P., additional, Wiegand, G., additional, and Wilichowski, E., additional

Published
2018
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26. The prospect for cytokine based therapeutic strategies in rheumatoid arthritis

Author

Campion, Giles V.

Subjects
Immune response -- Regulation, Cytokines -- Physiological aspects, Tumor necrosis factor -- Physiological aspects, Rheumatoid arthritis -- Care and treatment, Interleukins -- Physiological aspects, Health
Abstract

Research into the physiology of the immune system may lead to new kinds of therapies for rheumatoid arthritis. Interleukins (ILs), macrophages and tumor necrosis factor (TNF) are all components of the human body's immune system. Rheumatoid arthritis is an autoimmune disorder in which parts of the joints and the connective tissue become inflamed, swollen and damaged. Autoimmune disorders develop when the natural immune response, designed to attack invading organisms, attack the host. Understanding the genetic structure of the cells involved in the immune system and their role in inflammation can lead to the creation of antibodies to those components. Such monoclonal antibodies could be used to treat arthritis. Cytokines can be attacked by developing antagonists that can act at different points in the immune process. Abnormal cytokines and TNF have been related to certain illnesses and the level of cytokines may be related to the severity of the illness.

Published
1994

27. Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study

Author

Goemans, Nathalie M., primary, Tulinius, Már, additional, van den Hauwe, Marleen, additional, Kroksmark, Anna-Karin, additional, Buyse, Gunnar, additional, Wilson, Rosamund J., additional, van Deutekom, Judith C., additional, de Kimpe, Sjef J., additional, Lourbakos, Afrodite, additional, and Campion, Giles, additional

Published
2016
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28. Placebo‐controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy.

Author

McDonald, Craig M., Wong, Brenda, Flanigan, Kevin M., Wilson, Rosamund, de Kimpe, Sjef, Lourbakos, Afrodite, Lin, Zhengning, Campion, Giles, and the DEMAND V study group

Subjects
PLACEBOS, TREATMENT of Duchenne muscular dystrophy, PHARMACOKINETICS, PULMONARY function tests, DRUG efficacy
Abstract

Abstract: Objective: This double‐blind, randomized, placebo‐controlled Phase 2 study (NCT01462292) assessed the 24‐week efficacy, safety, tolerability, and pharmacokinetics of two different subcutaneous drisapersen doses, and the 24‐week off‐dose persistent effect, in ambulant Duchenne muscular dystrophy (DMD) patients. Methods: Male DMD patients (≥5 years; time to rise from floor ≤15 s) were randomized to drisapersen 3 mg/kg/week, 6 mg/kg/week or placebo. The primary efficacy endpoint was change from baseline in 6‐minute walking distance (6MWD) at week 24. Secondary endpoints included changes in timed function tests, muscle strength, and pulmonary function tests. Results: Fifty‐one patients were randomized to placebo (N = 16), drisapersen 3 mg/kg/week (N = 17) or 6 mg/kg/week (N = 18). All but 2 patients had baseline rise from floor time <7 s. This study was exploratory and not prospectively powered; however, a difference in mean 6MWD versus placebo in favor of drisapersen 6 mg/kg/week was observed at week 24 (27.1 m; P = 0.069) and maintained 24 weeks off‐treatment (27.9 m; P = 0.177). The 3 mg/kg/week group showed no statistically significant difference in mean 6MWD versus placebo. For some secondary endpoints, a more positive response in favor of drisapersen 6 mg/kg/week compared to placebo was shown. Drisapersen had a long half‐life with steady state reached after approximately 36 weeks. Most common adverse events in both drisapersen groups were related to injection site reactions and subclinical proteinuria. Interpretation: Drisapersen 6 mg/kg/week for 24 weeks resulted in a treatment benefit in 6MWD, largely maintained 24 weeks off‐treatment. This study provided insights for further studies to optimize dosage regimen. [ABSTRACT FROM AUTHOR]

Published
2018
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36. A Sensitive, Reproducible and Objective Immunofluorescence Analysis Method of Dystrophin in Individual Fibers in Samples from Patients with Duchenne Muscular Dystrophy

Author

Beekman, Chantal, primary, Sipkens, Jessica A., additional, Testerink, Janwillem, additional, Giannakopoulos, Stavros, additional, Kreuger, Dyonne, additional, van Deutekom, Judith C., additional, Campion, Giles V., additional, de Kimpe, Sjef J., additional, and Lourbakos, Afrodite, additional

Published
2014
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37. Exon skipping for DMD

Author

Aartsma-Rus, Annemieke, primary, Verschuuren, Jan JGM, additional, Campion, Giles V, additional, van Ommen, Gert-jan B, additional, and van Deutekom, Judith CT, additional

Published
2012
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38. Author's reply

Author

Campion, Giles V

Subjects
Matters Arising
Published
1994

39. Generalised osteoarthritis: a hormonally mediated disease

Author

Spector, Tim D. and Campion, Giles D.

Subjects
Osteoarthritis -- Causes of, Estrogen -- Physiological aspects, Menopause -- Physiological aspects, Health
Abstract

Osteoarthritis (OA), a long-term joint disease, is characterized by destruction of the cartilage in the joint, and overgrowth, malformation, and impaired function of the bone. The prevalence of OA is high, affecting up to 70 percent of persons aged 90 years or over. Among women, OA affects multiple joints and is more severe than in men. Because OA is prevalent among middle-aged women, it was suggested that there may be a relation between menopause and the occurrence of OA. Generalized OA has also been associated with obesity, the presence of fibroids (fiber-like growths), and bleeding of the uterus, but not with osteoporosis (bone loss). This suggests that OA is related to excessive amounts of the female hormone estrogen. Women with high estrogen levels are at risk of developing OA, particularly during menopause, when estrogen levels are relatively high when compared with progesterone levels. In animal models of OA, the joint lesions became worse with estrogen but improved with tamoxifen, an agent that blocks the actions of estrogen. It is thought that estrogen may interfere with the processes of bone formation and dissolution, and with cartilage turnover, which is the balance between the production and breakdown of cartilage tissue. Thus, in addition to the evolutionary, mechanical, and genetic causes of OA, hormonal factors may also influence the development of this joint disease. (Consumer Summary produced by Reliance Medical Information, Inc.)

Published
1989

40. Dystrophin quantification: Biological and translational research implications.

Author

Anthony, Karen, Arechavala-Gomeza, Virginia, Taylor, Laura E, Vulin, Adeline, Kaminoh, Yuuki, Torelli, Silvia, Feng, Lucy, Janghra, Narinder, Bonne, Gisèle, Beuvin, Maud, Barresi, Rita, Henderson, Matt, Laval, Steven, Lourbakos, Afrodite, Campion, Giles, Straub, Volker, Voit, Thomas, Sewry, Caroline A, Morgan, Jennifer E, and Flanigan, Kevin M

Published
2014
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41. Dystrophin quantification.

Author

Anthony, Karen, Arechavala-Gomeza, Virginia, Taylor, Laura E., Vulin, Adeline, Yuuki Kaminoh, Torelli, Silvia, Feng, Lucy, Janghra, Narinder, Bonne, Gisèle, Beuvin, Maud, Barresi, Rita, Henderson, Matt, Laval, Steven, Lourbakos, Afrodite, Campion, Giles, Straub, Volker, Voit, Thomas, Sewry, Caroline A., Morgan, Jennifer E., and Flanigan, Kevin M.

Published
2014

42. Clinical Manifestations of Human Parvovirus B19 in Adults

Author

Woolf, Anthony D., Campion, Giles V., Chishick, Alice, Wise, Stephen, Cohen, Bernard J., Klouda, Peter T., Caul, Owen, and Dieppe, Paul A.

Abstract

• Human parvovirus B19 has been associated with various clinical effects in a number of uncontrolled reports. To define the usual manifestations of B19 infection in adults and the factors that influence them we present a clinicoepidemiological study of an outbreak of B19 infection centered on a junior school. Four hundred fifty-three of 475 adults in this community were interviewed and blood was obtained for serological diagnosis. Fifty-four cases of recent infection were identified and were HLA typed. Fourteen of the cases were asymptomatic; 32 had an influenzalike illness; 23 a rash; and 26 an acute-onset polyarthropathy that was more common in women and lasted for up to 7 months. HLA-A, -B, and -C antigen frequencies were similar to a local control population and showed no association with symptoms except that HLA-DR1 was absent in those with persistent arthropathy.(Arch Intern Med. 1989;149:1153-1156)

Published
1989
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