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Use of the patient-reported outcomes measurement information system (PROMIS®) to assess late-onset Pompe disease severity.
Published in:
Journal of Patient-Reported Outcomes, 2020, v. 4, n. 1, p. N.PAG, doi. 10.1186/s41687-020-00245-2
By:
- Harfouche, Melodi;
- Kishnani, Priya S.;
- Krusinska, Eva;
- Gault, Jamie;
- Sitaraman, Sheela;
- Sowinski, Amanda;
- Katz, Irina;
- Austin, Stephanie;
- Goldstein, Margi;
- Mulberg, Andrew E.
Publication type:
Article
Reduced expression of the cystic fibrosis transmembrane conductance regulator gene in the hypothalamus of patients with Alzheimer's disease.
Published in:
Journal of Alzheimer's Disease, 2003, v. 5, n. 6, p. 455
By:
- Lahousse, Stephanie A.;
- Stopa, Edward G.;
- Mulberg, Andrew E.;
- De la Monte, Suzanne M.
Publication type:
Article
Regulatory strategies for rare diseases under current global regulatory statutes: a discussion with stakeholders.
Published in:
2019
By:
- Mulberg, Andrew E.;
- Bucci-Rechtweg, Christina;
- Giuliano, Joseph;
- Jacoby, David;
- Johnson, Franklin K.;
- Liu, Qing;
- Marsden, Deborah;
- McGoohan, Scott;
- Nelson, Robert;
- Patel, Nita;
- Romero, Klaus;
- Sinha, Vikram;
- Sitaraman, Sheela;
- Spaltro, John;
- Kessler, Vivian
Publication type:
journal article
Research Agenda for Pediatric Gastroenterology, Hepatology and Nutrition: Cystic Fibrosis and Pancreatic Diseases: Report of the North American Society for Pediatric Gastroenterology, Hepatology and Nutrition for the Children's Digestive Health and Nutrition Foundation
Published in:
Journal of Pediatric Gastroenterology & Nutrition, 2002, v. 35, p. S258
By:
- Lowe, Mark E.;
- Ameen, Nadia;
- Freedman, Stephen;
- Mulberg, Andrew E.;
- Werlin, Steven L.
Publication type:
Article
Rethinking growth failure in Alagille syndrome: the role of dietary intake and steatorrhea.
Published in:
2002
By:
- Rovner, Alisha J.;
- Schall, Joan I.;
- Jawad, Abbas F.;
- Piccoli, David A.;
- Stallings, Virginia A.;
- Mulberg, Andrew E.;
- Zemel, Babette S.
Publication type:
journal article
Fatal cholestatic hepatitis and multisystem failure associated with nitrofurantoin.
Published in:
1993
By:
- Mulberg, Andrew E.;
- Bell, Louis M.;
- Mulberg, A E;
- Bell, L M
Publication type:
journal article
Pediatric Intestinal Failure-Associated Liver Disease: Challenges in Identifying Clinically Relevant Biomarkers.
Published in:
2018
By:
- Gura, Kathleen M.;
- Mulberg, Andrew E.;
- Mitchell, Paul D.;
- Yap, John;
- Kim, Clara Y.;
- Chen, Minjun;
- Potemkin, Alexis;
- Puder, Mark
Publication type:
journal article
Correction to: Sharing is caring: a call for a new era of rare disease research and development.
Published in:
Orphanet Journal of Rare Diseases, 2023, v. 18, n. 1, p. 1, doi. 10.1186/s13023-023-02613-9
By:
- Denton, Nathan;
- Mulberg, Andrew E.;
- Molloy, Monique;
- Charleston, Samantha;
- Fajgenbaum, David C.;
- Marsh, Eric D.;
- Howard, Paul
Publication type:
Article
Sharing is caring: a call for a new era of rare disease research and development.
Published in:
2022
By:
- Denton, Nathan;
- Mulberg, Andrew E.;
- Molloy, Monique;
- Charleston, Samantha;
- Fajgenbaum, David C.;
- Marsh, Eric D.;
- Howard, Paul
Publication type:
journal article
Data silos are undermining drug development and failing rare disease patients.
Published in:
2021
By:
- Denton, Nathan;
- Molloy, Monique;
- Charleston, Samantha;
- Lipset, Craig;
- Hirsch, Jonathan;
- Mulberg, Andrew E.;
- Howard, Paul;
- Marsh, Eric D.
Publication type:
journal article
Exposure Matching for Extrapolation of Efficacy in Pediatric Drug Development.
Published in:
Journal of Clinical Pharmacology, 2016, v. 56, n. 11, p. 1326, doi. 10.1002/jcph.744
By:
- Mulugeta, Yeruk;
- Barrett, Jeffrey S.;
- Nelson, Robert;
- Eshete, Abel Tilahun;
- Mushtaq, Alvina;
- Yao, Lynne;
- Glasgow, Nicole;
- Mulberg, Andrew E.;
- Gonzalez, Daniel;
- Green, Dionna;
- Florian, Jeffry;
- Krudys, Kevin;
- Seo, Shirley;
- Kim, Insook;
- Chilukuri, Dakshina;
- Burckart, Gilbert J.
Publication type:
Article
Turnover of the cystic fibrosis transmembrane conductance regulator (CFTR): Slow degradation of wild-type and ΔF508 CFTR in surface membrane preparations of immortalized airway epithelial cells.
Published in:
Journal of Cellular Physiology, 1996, v. 168, n. 2, p. 373, doi. 10.1002/(SICI)1097-4652(199608)168:2<373::AID-JCP16>3.0.CO;2-4
By:
- Wei, Xiaofang;
- Eisman, Robin;
- Xu, Jin;
- Harsch, Alan D.;
- Mulberg, Andrew E.;
- Bevins, Charles L.;
- Glick, Mary Catherine;
- Scanlin, Thomas F.
Publication type:
Article
Expediting Drug Development for Pediatric Inflammatory Bowel Disease: A Discussion With Stakeholders.
Published in:
Journal of Pediatric Gastroenterology & Nutrition, 2018, v. 66, n. 1, p. 95, doi. 10.1097/MPG.0000000000001824
By:
- Mulberg, Andrew E.;
- Bousvaros, Athos;
- Colletti, Richard;
- Hyams, Jeffrey;
- Lee, Kerry Jo;
- Musaus, Joachim;
- Robinson, Anne M.;
- Rosh, Joel;
- Saeed, Shehzad;
- Taylor-Zapata, Perdita;
- Zuckerman, Eric;
- Winter, Harland S.
Publication type:
Article
Pediatric Crohn Disease Clinical Outcome Assessments and Biomarkers: Current State and Path Forward for Global Collaboration.
Published in:
2017
By:
- Sun, Haihao;
- Vesely, Richard;
- Lee, Kerry Jo;
- Klein, Agnes;
- Ikima, Mutsuhiro;
- Mulberg, Andrew E.;
- International Inflammatory Bowel Disease (i-IBD) Working Group
Publication type:
journal article
Well-Defined and Reliable Clinical Outcome Assessments for Pediatric Crohn Disease: A Critical Need for Drug Development.
Published in:
Journal of Pediatric Gastroenterology & Nutrition, 2015, v. 60, n. 6, p. 729, doi. 10.1097/MPG.0000000000000793
By:
- Haihao Sun;
- Papadopoulos, Elektra J.;
- Hyams, Jeffrey S.;
- Griebel, Donna;
- Lee, Jessica J.;
- Tomaino, Juli;
- Mulberg, Andrew E.
Publication type:
Article
Steps toward harmonization for clinical development of medicines in pediatric ulcerative colitis-a global scientific discussion, part 2: data extrapolation, trial design, and pharmacokinetics.
Published in:
2014
By:
- Sun, Haihao;
- Vesely, Richard;
- Nelson, Robert M;
- Taminiau, Jan;
- Szitanyi, Peter;
- Isaac, Maria;
- Klein, Agnes;
- Uzu, Shinobu;
- Griebel, Donna;
- Mulberg, Andrew E
Publication type:
Journal Article
Alternate endpoints and clinical outcome assessments in pediatric ulcerative colitis registration trials.
Published in:
2014
By:
- Sun, Haihao;
- Lee, Jessica J;
- Papadopoulos, Elektra J;
- Lee, Catherine S;
- Nelson, Robert M;
- Sachs, Hari C;
- Rodriguez, William J;
- Mulberg, Andrew E
Publication type:
Journal Article
Body composition of children aged 1 to 12 years with biliary atresia or Alagille syndrome.
Published in:
2005
By:
- Arvay, Jessica L;
- Zemel, Babette S;
- Gallagher, Paul R;
- Rovner, Alisha J;
- Mulberg, Andrew E;
- Stallings, Virgina A;
- Haber, Barbara A
Publication type:
journal article
Deficits in size-adjusted bone mass in children with Alagille syndrome.
Published in:
2005
By:
- Olsen, Irene E.;
- Ittenbach, Richard F.;
- Rovner, Alisha J.;
- Leonard, Mary B.;
- Mulberg, Andrew E.;
- Stallings, Virginia A.;
- Piccoli, David A.;
- Zemel, Babette S.
Publication type:
journal article
Extrapolation of Adult Efficacy to Pediatric Patients With Chemotherapy‐Induced Nausea and Vomiting.
Published in:
Journal of Clinical Pharmacology, 2020, v. 60, n. 6, p. 775, doi. 10.1002/jcph.1577
By:
- Momper, Jeremiah D.;
- Heinrichs, M. Tobias;
- Krudys, Kevin;
- Griebel, Donna;
- Kumar, Shaun;
- Kim, Insook;
- Mehrotra, Nitin;
- Mulberg, Andrew E.;
- Garimella, Narayana;
- Nelson, Robert;
- Reaman, Gregory;
- Sinha, Vikram;
- Yao, Lynne;
- Zineh, Issam;
- Burckart, Gilbert;
- Sachs, Hari;
- Mulugeta, Yeruk
Publication type:
Article
FABry Disease Patient-Reported Outcome-GastroIntestinal (FABPRO-GI): A new Fabry disease-specific gastrointestinal outcomes instrument.
Published in:
Quality of Life Research, 2021, v. 30, n. 10, p. 2983, doi. 10.1007/s11136-021-02847-9
By:
- Shields, Alan L.;
- Lamoureux, Roger E.;
- Taylor, Fiona;
- Barth, Jay A.;
- Mulberg, Andrew E.;
- Kessler, Vivian;
- Skuban, Nina
Publication type:
Article
Proceedings From FDA/A.S.P.E.N. Public Workshop.
Published in:
JPEN Journal of Parenteral & Enteral Nutrition, 2015, v. 39, n. 7, p. 768, doi. 10.1177/0148607114560825
By:
- Teitelbaum, Daniel H.;
- Guenter, Peggi;
- Griebel, Donna;
- Abrams, Steven A.;
- Bark, Staffan;
- Baker, Mary;
- Berry, Karyn L.;
- Bistrian, Bruce R.;
- Brenna, J. Thomas;
- Bonnot, Denis;
- Carpentier, Yvon A.;
- Deckelbaum, Richard J.;
- Hise, Mary;
- Koletzko, Berthold;
- Mirtallo, Jay M.;
- Mulberg, Andrew E.;
- O’Reilly, Randall C.;
- Shaffer, Jonathan;
- von Kleist, Elke;
- Zaloga, Gary P.
Publication type:
Article
Intestinal cytokines in children with pervasive developmental disorders
Published in:
American Journal of Gastroenterology (Springer Nature), 2003, v. 98, n. 8, p. 1777, doi. 10.1111/j.1572-0241.2003.07593.x
By:
- DeFelice, Magee L.;
- Ruchelli, Eduardo D.;
- Markowitz, Jonathan E.;
- Strogatz, Melissa;
- Reddy, Krishna P.;
- Kadivar, Khadijeh;
- Mulberg, Andrew E.;
- Brown, Kurt A.
Publication type:
Article

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