Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy
Finkel, Richard S, Flanigan, Kevin M, Wong, Brenda, Bönnemann, Carsten, Sampson, Jacinda, Sweeney, H Lee, Reha, Allen, Northcutt, Valerie J, Elfring, Gary, Barth, Jay, Peltz, Stuart W
Published in PloS one (11.12.2013)
Published in PloS one (11.12.2013)
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The challenges of living with and managing epidermolysis bullosa: insights from patients and caregivers
Bruckner, Anna L, Losow, Michael, Wisk, Jayson, Patel, Nita, Reha, Allen, Lagast, Hjalmar, Gault, Jamie, Gershkowitz, Jayne, Kopelan, Brett, Hund, Michael, Murrell, Dedee F
Published in Orphanet journal of rare diseases (03.01.2020)
Published in Orphanet journal of rare diseases (03.01.2020)
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THE 6-minute walk test and other endpoints in Duchenne muscular dystrophy: Longitudinal natural history observations over 48 weeks from a multicenter study
Mcdonald, Craig M., Henricson, Erik K., Abresch, R. Ted, Florence, Julaine M., Eagle, Michelle, Gappmaier, Eduard, Glanzman, Allan M., Spiegel, Robert, Barth, Jay, Elfring, Gary, Reha, Allen, Peltz, Stuart
Published in Muscle & nerve (01.09.2013)
Published in Muscle & nerve (01.09.2013)
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The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: Reliability, concurrent validity, and minimal clinically important differences from a multicenter study
McDonald, Craig M., Henricson, Erik K., Abresch, R. Ted, Florence, Julaine, Eagle, Michelle, Gappmaier, Eduard, Glanzman, Allan M., Spiegel, Robert, Barth, Jay, Elfring, Gary, Reha, Allen, Peltz, Stuart W.
Published in Muscle & nerve (01.09.2013)
Published in Muscle & nerve (01.09.2013)
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The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy
McDonald, Craig M., Henricson, Erik K., Han, Jay J., Abresch, R. Ted, Nicorici, Alina, Elfring, Gary L., Atkinson, Leone, Reha, Allen, Hirawat, Samit, Miller, Langdon L.
Published in Muscle & nerve (01.04.2010)
Published in Muscle & nerve (01.04.2010)
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Efficacy and tolerability of the investigational topical cream SD-101 (6% allantoin) in patients with epidermolysis bullosa: a phase 3, randomized, double-blind, vehicle-controlled trial (ESSENCE study)
Paller, Amy S, Browning, John, Nikolic, Milos, Bodemer, Christine, Murrell, Dedee F, Lenon, Willistine, Krusinska, Eva, Reha, Allen, Lagast, Hjalmar, Barth, Jay A
Published in Orphanet journal of rare diseases (23.06.2020)
Published in Orphanet journal of rare diseases (23.06.2020)
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Wound closure in epidermolysis bullosa: data from the vehicle arm of the phase 3 ESSENCE Study
Murrell, Dedee F., Paller, Amy S., Bodemer, Christine, Browning, John, Nikolic, Milos, Barth, Jay A., Lagast, Hjalmar, Krusinska, Eva, Reha, Allen
Published in Orphanet journal of rare diseases (21.07.2020)
Published in Orphanet journal of rare diseases (21.07.2020)
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Corticosteroids in Duchenne muscular dystrophy: Major variations in practice
Griggs, Robert C., Herr, Barbara E., Reha, Allen, Elfring, Gary, Atkinson, Leone, Cwik, Valerie, Mccoll, Elaine, Tawil, Rabi, Pandya, Shree, McDermott, Michael P., Bushby, Kate
Published in Muscle & nerve (01.07.2013)
Published in Muscle & nerve (01.07.2013)
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Ataluren (PTC124) Induces Cystic Fibrosis Transmembrane Conductance Regulator Protein Expression and Activity in Children with Nonsense Mutation Cystic Fibrosis
SERMET-GAUDELUS, Isabelle, DE BOECK, Kris, MILLERS, Nilsen L, CONSTANTINE, Scott, REHA, Allen, AJAYI, Temitayo, ELFRING, Gary L, MILLERS, Langdon L, CASIMIR, Georges J, VERMEULEN, François, LEAL, Teresinha, MOGENET, Agnès, ROUSSEL, Delphine, FRITSCH, Janine, HANSSENS, Laurence, HIRAWAT, Samit
Published in American journal of respiratory and critical care medicine (15.11.2010)
Published in American journal of respiratory and critical care medicine (15.11.2010)
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Single-dose AAV9-CLN6 gene transfer stabilizes motor and language function in CLN6-type Batten disease: Interim results from the first clinical gene therapy trial
de los Reyes, Emily, Meyer, Kathrin, Lehwald, Lenora, Albright, Charles, Castelli, Jeff, Jiang, Hai, Reha, Allen, Barth, Jay
Published in Molecular genetics and metabolism (01.02.2020)
Published in Molecular genetics and metabolism (01.02.2020)
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The 6-minute walk test in Duchenne/Becker muscular dystrophy: Longitudinal observations
MCDonald, Craig M., Henricson, Erik K., Han, Jay J., Abresch, R. Ted, Nicorici, Alina, Atkinson, Leone, Elfring, Gary L., Reha, Allen, Miller, Langdon L.
Published in Muscle & nerve (01.12.2010)
Published in Muscle & nerve (01.12.2010)
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Journal Article
Percent-predicted 6-minute walk distance in duchenne muscular dystrophy to account for maturational influences
Henricson, Erik, Abresch, Richard, Han, Jay J, Nicorici, Alina, Goude Keller, Erica, Elfring, Gary, Reha, Allen, Barth, Jay, McDonald, Craig M
Published in PLoS currents (25.01.2012)
Published in PLoS currents (25.01.2012)
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Ambulatory quantitative waking and sleeping cough assessment in patients with cystic fibrosis
Kerem, Eitan, Wilschanski, Michael, Miller, Nilsen L, Pugatsch, Thea, Cohen, Tali, Blau, Hannah, Rivlin, Joseph, Shoseyov, David, Reha, Allen, Constantine, Scott, Ajayi, Temitayo, Hirawat, Samit, Elfring, Gary L, Peltz, Stuart W, Miller, Langdon L
Published in Journal of cystic fibrosis (01.05.2011)
Published in Journal of cystic fibrosis (01.05.2011)
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Journal Article
Phase 2a Study of Ataluren-Mediated Dystrophin Production in Patients with Nonsense Mutation Duchenne Muscular Dystrophy: e81302
Finkel, Richard S, Flanigan, Kevin M, Wong, Brenda, Bonnemann, Carsten, Sampson, Jacinda, Sweeney, H Lee, Reha, Allen, Northcutt, Valerie J, Elfring, Gary, Barth, Jay
Published in PloS one (01.12.2013)
Published in PloS one (01.12.2013)
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Abstract #294: New Clinical Experience With Pasireotide (Som230), the Novel Multiligand Somatostatin Analogue
Glusman, Joan, Reha, Allen, McBride, Karen, Schmid, Herbert
Published in Endocrine practice (01.03.2006)
Published in Endocrine practice (01.03.2006)
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