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

Results: 34

ASCPT News.

Published in:: Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1371, doi. 10.1002/cpt.3475
Publication type:: Article

HIGHLIGHTED ARTICLES.

Published in:: Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1369, doi. 10.1002/cpt.3476
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

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

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

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

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

In This Issue.

Published in:: Clinical Pharmacology & Therapeutics, 2024, v. 116, n. 6, p. 1361, doi. 10.1002/cpt.3473
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

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

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

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

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

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

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

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

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

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