Works matching IS 00099236 AND DT 2024 AND VI 116 AND IP 6

Results: 34

HIGHLIGHTED ARTICLES.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1369, doi. 10.1002/cpt.3476
Publication type:
Article
ASCPT News.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1371, doi. 10.1002/cpt.3475
Publication type:
Article
Therapeutic Orphan No More: Role for Clinical Pharmacology and Translational Science in Developing Therapeutics for Rare and Neglected Diseases.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1363, doi. 10.1002/cpt.3474
By:
- Ramamoorthy, Anuradha;
- Younis, Islam;
- Wen, Ya‐Feng;
- Mehanna, Mai;
- Giacomini, Kathleen M.;
- van der Graaf, Piet H.
Publication type:
Article
In This Issue.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1361, doi. 10.1002/cpt.3473
Publication type:
Article
First‐in‐Human Evaluation of Safety, Pharmacokinetics and Muscle Glycogen Lowering of a Novel Glycogen Synthase 1 Inhibitor for the Treatment of Pompe Disease.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1580, doi. 10.1002/cpt.3470
By:
- Ullman, Julie C.;
- Dick, Ryan A.;
- Linzner, Daniela;
- Minga, Todd;
- Tep, Samnang;
- Satterfield, Terrence F.;
- Xi, Yannan;
- Beattie, David T.;
- Marmon, Tonya;
- Neutel, Joel M.;
- Chung, Bernard;
- Leeds, Janet M.;
- Noonberg, Sarah B.;
- Green, Eric M.;
- Bernstein, Harold S.
Publication type:
Article
Model‐Informed Approach to Recommend Burosumab Dosing Regimens for Pediatric and Adult Patients With the Ultrarare Disease Tumor‐Induced Osteomalacia.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1606, doi. 10.1002/cpt.3468
By:
- Hruska, Matthew W.;
- Sid‐Otmane, Lamia;
- Gosselin, Nathalie H.;
- Quattrocchi, Emilia;
- Lee, Sun Ku;
- Mascelli, Mary Ann;
- Mehta, Krina;
- Jan de Beur, Suzanne M.;
- Marsteller, Douglas
Publication type:
Article
Longitudinal Analysis of Natural History Progression of Rare and Ultra‐Rare Cerebellar Ataxias Using Item Response Theory.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1593, doi. 10.1002/cpt.3466
By:
- Hamdan, Alzahra;
- Hendrickx, Niels;
- Hooker, Andrew C.;
- Chen, Xiaomei;
- Comets, Emmanuelle;
- Traschütz, Andreas;
- Schüle, Rebecca;
- Heussen, Nicole Maria;
- Hilgers, Ralf‐Dieter;
- Klockgether, Thomas;
- Ryeznik, Yevgen;
- Sverdlov, Oleksandr;
- Mentré, France;
- Synofzik, Matthis;
- Karlsson, Mats O.
Publication type:
Article
Quantitative Systems Pharmacology Models: Potential Tools for Advancing Drug Development for Rare Diseases.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1442, doi. 10.1002/cpt.3451
By:
- Neves‐Zaph, Susana;
- Kaddi, Chanchala
Publication type:
Article
Infrequent Resolution of Vaso‐Occlusive Crises in Routine Clinical Care Among Patients Mimicking the Exa‐Cel Trial Population: A Cohort Study of Medicaid Enrollees.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1572, doi. 10.1002/cpt.3449
By:
- Mahesri, Mufaddal;
- Lee, Su Been;
- Levin, Raisa;
- Imren, Suzan;
- Zhang, Lanju;
- Beukelman, Timothy;
- Titievsky, Lina;
- Desai, Rishi J.
Publication type:
Article
What Is Rare and What Is Orphan? A Guide to the Regulatory Terminology.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1395, doi. 10.1002/cpt.3446
By:
- Larsson, Kristina
Publication type:
Article
External Controls to Study Treatment Effects in Rare Diseases: Challenges and Future Directions.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1521, doi. 10.1002/cpt.3443
By:
- Weberpals, Janick;
- Wang, Shirley V.
Publication type:
Article
Extrapolation of Upadacitinib Efficacy in Juvenile Idiopathic Arthritis Leveraging Pharmacokinetics, Exposure–Response Models, and Real‐World Patient Data.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1635, doi. 10.1002/cpt.3441
By:
- Qian, Yuli;
- Schlachter, Louisa;
- Eckert, Doerthe;
- Stodtmann, Sven;
- Shmagel, Anna;
- Peng, Yi;
- Liu, Wei;
- Mohamed, Mohamed‐Eslam F.
Publication type:
Article
Rare Diseases Linked to Mutations in Vitamin Transporters Expressed in the Human Blood–Brain Barrier.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1513, doi. 10.1002/cpt.3433
By:
- Yee, Sook Wah;
- Wang, Joanne;
- Giacomini, Kathleen M.
Publication type:
Article
Diagnosis and Management of Parathyroid Carcinoma.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1496, doi. 10.1002/cpt.3432
By:
- Lazzaro, Alexander;
- Zhao, Grace;
- Kulke, Matthew
Publication type:
Article
First‐in‐Human Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of BMS‐986308: A Renal Outer Medullary Potassium Channel Inhibitor.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1627, doi. 10.1002/cpt.3430
By:
- Soleman, Sharif I.;
- Maya, Juan;
- Levesque, Paul;
- Mohammad, Atif;
- Christopher, Lisa;
- Schumacher, Justin;
- Nanduri, Aparna;
- Sivakumar, Pitchumani;
- Kozinn, Marc;
- Costet, Philippe;
- Wang, Chang;
- Richter, Jeremy;
- Hawthorne, Dara;
- Bui, Anh;
- Rao, Veena S.;
- Dickerson, Daniel;
- Testani, Jeffrey;
- Ramírez‐Valle, Francisco;
- Baribaud, Frédéric;
- Murthy, Bindu
Publication type:
Article
The Rare Disease Moonshot: Paradigms Shift, Translational Medicine, and Regulatory Science for the World's Rarest Conditions.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1387, doi. 10.1002/cpt.3428
By:
- Ollivier, Cécile;
- Corriol‐Rohou, Solange;
- del Álamo, Marta;
- Favresse, Roseline;
- Kostenzer, Johanna;
- Boudes, Mathieu;
- Ussi, Anton E.;
- Viel, Klaus;
- Linden, R. Michael;
- Chlebus, Magda
Publication type:
Article
The Promise of Health for All: A Collaborative Model to Close Treatment Gaps for Neglected Diseases.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1385, doi. 10.1002/cpt.3427
By:
- Attipoe, Delali
Publication type:
Article
Model‐Informed Approaches to Optimizing Therapeutics in the African Patient Populations.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1391, doi. 10.1002/cpt.3425
By:
- Mulubwa, Mwila;
- Chibale, Kelly
Publication type:
Article
Pediatric Rare Diseases Development in the Pharmaceutical Industry: An International Consortium for Innovation and Quality in Pharmaceutical Development, Clinical Pharmacology Leadership Group‐Pediatrics Working Group, Rare Diseases Subteam Whitepaper Examining the Current Landscape and Recommendations for the Future
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1433, doi. 10.1002/cpt.3422
By:
- Krishna, Rajesh;
- Sebastien, Bernard;
- Corriol‐Rohou, Solange;
- Cheung, S. Y. Amy;
- Liu, Jing;
- Suryawanshi, Satyendra
Publication type:
Article
Progress in Pharmacometrics Implementation and Regulatory Integration in Africa: A Systematic Review.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1525, doi. 10.1002/cpt.3415
By:
- Ndzamba, Bonginkosi S'fiso;
- Egieyeh, Samuel;
- Fasinu, Pius
Publication type:
Article
Ongoing Pharmacological Developments in SLC6A1 Research & Treatment.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1381, doi. 10.1002/cpt.3413
By:
- DeLeeuw, Melissa B.;
- Tiller, Jacob;
- Kang, Jing‐Qiong;
- Freed, Amber
Publication type:
Article
Developmental Expression of Drug Transporters and Conjugating Enzymes Involved in Enterohepatic Recycling: Implication for Pediatric Drug Dosing.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1615, doi. 10.1002/cpt.3409
By:
- Thakur, Aarzoo;
- Subash, Sandhya;
- Ahire, Deepak;
- Prasad, Bhagwat
Publication type:
Article
Getting the Dose Right in Drug Development for Rare Diseases: Barriers and Enablers.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1412, doi. 10.1002/cpt.3407
By:
- Ahmed, Mariam A.;
- Krishna, Rajesh;
- Rayad, Noha;
- Albusaysi, Salwa;
- Mitra, Amitava;
- Shang, Elizabeth;
- Hon, Yuen Yi;
- AbuAsal, Bilal;
- Bakhaidar, Rana;
- Roman, Youssef M.;
- Bhattacharya, Indranil;
- Cloyd, James;
- Patel, Munjal;
- Kartha, Reena V.;
- Younis, Islam R.
Publication type:
Article
Assessing Post‐Marketing Requirements for Orphan Drugs: A Cross‐Sectional Analysis of FDA and EMA Oversight.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1560, doi. 10.1002/cpt.3397
By:
- Yu, Jae Hyeon;
- Lee, Sangwon;
- Kim, Yoon Jung;
- Kim, Won Young;
- Lee, Min Jung;
- Kim, Yun
Publication type:
Article
From Challenge to Opportunity: How Shwachman–Diamond Syndrome Became a Promising Target for Therapy Development.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1377, doi. 10.1002/cpt.3393
By:
- Hars, Eszter S.;
- McReynolds, Lisa J.
Publication type:
Article
Impact of Priority Review Voucher Eligibility on Research and Development of Medical Countermeasures.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1554, doi. 10.1002/cpt.3388
By:
- Wang, Stephanie;
- Hahn, Georg;
- Kesselheim, Aaron S.
Publication type:
Article
Integrated Applied Clinical Pharmacology in the Advancement of Rare and Ultra‐Rare Disease Therapeutics.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1485, doi. 10.1002/cpt.3382
By:
- Ryder, Steven
Publication type:
Article
New Horizons of Model Informed Drug Development in Rare Diseases Drug Development.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1398, doi. 10.1002/cpt.3366
By:
- Mitra, Amitava;
- Tania, Nessy;
- Ahmed, Mariam A.;
- Rayad, Noha;
- Krishna, Rajesh;
- Albusaysi, Salwa;
- Bakhaidar, Rana;
- Shang, Elizabeth;
- Burian, Maria;
- Martin‐Pozo, Michelle;
- Younis, Islam R.
Publication type:
Article
Systemic Treatment of Body‐Wide Duchenne Muscular Dystrophy Symptoms.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1472, doi. 10.1002/cpt.3363
By:
- Konieczny, Patryk
Publication type:
Article
Racial and Ethnic Disparities in Antihypertensive Medication Prescribing Patterns and Effectiveness.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1544, doi. 10.1002/cpt.3360
By:
- Goleva, Slavina B.;
- Williams, Ariel;
- Schlueter, David J.;
- Keaton, Jacob M.;
- Tran, Tam C.;
- Waxse, Bennett J.;
- Ferrara, Tracey M.;
- Cassini, Thomas;
- Mo, Huan;
- Denny, Joshua C.
Publication type:
Article
Attaining Equity of Access to Research: Perspective on Research in Pregnancy and Breastfeeding Following Dolores Shockley Lecture at ASCPT2024.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1506, doi. 10.1002/cpt.3344
By:
- Waitt, Catriona;
- Asiimwe, Simon Peter;
- Ojara, Francis Williams;
- Nakijoba, Ritah;
- Kawuma, Aida N;
- Castelnuovo, Barbara
Publication type:
Article
Advances in Clinical Therapies for Huntington's Disease and the Promise of Multi‐Targeted/Functional Drugs Based on Clinicaltrials.gov.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1452, doi. 10.1002/cpt.3341
By:
- Huang, Chunhui;
- Zheng, Xiao;
- Yan, Sen;
- Zhang, Zaijun
Publication type:
Article
Medications and Acute Hemolysis in G6PD‐Deficient Patients – A Real‐World Study.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1537, doi. 10.1002/cpt.3333
By:
- Gronich, Naomi;
- Rosh, Bar;
- Stein, Nili;
- Saliba, Walid
Publication type:
Article
Issue Information.
Published in:
Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1353, doi. 10.1002/cpt.2949
Publication type:
Article