Results 111 to 120 of about 3,868,024 (353)

Recommended Phase II Doses of Talquetamab in Patients With Relapsed/Refractory Multiple Myeloma From MonumenTAL‐1: Clinical Pharmacology Results

Clinical Pharmacology &Therapeutics, EarlyView.
Talquetamab is the first and only GPRC5D × CD3 bispecific antibody approved for relapsed/refractory multiple myeloma (RRMM). In the phase I/II MonumenTAL‐1 study, overall response rates (ORRs) were > 66% in patients with RRMM treated with subcutaneous talquetamab at the recommended phase II doses (RP2Ds): 0.4 mg/kg weekly and 0.8 mg/kg every other week.
Jue Gong, Jie Zhou, Dongfen Yuan, Xuewen Ma, Deeksha Vishwamitra, Brandi Hilder, Tara J. Masterson, Jaszianne Tolbert, Thomas Renaud, Christoph Heuck, Colleen Kane, Mahesh N. Samtani, Suzette Girgis, Nahor Haddish‐Berhane, Jesus Berdeja, Amrita Krishnan, Daniele Ouellet   +16 more
wiley   +1 more source

Successful private–public funding of paediatric medicines research: lessons from the EU programme to fund research into off-patent medicines [PDF]

, 2014
The European Paediatric Regulation mandated the European Commission to fund research on off-patent medicines with demonstrated therapeutic interest for children.
Baiardi, P, Bonifazi, D, Bonifazi, F, Ceci, A, Chiron, C, Davies, E H, Eichler, I, Felisi, M, Giannuzzi, V, Giaquinto, C, Jacqz-Aigrain, E, Neubert, A, on behalf of the GRiP Consortium,, Pressler, R, Rabe, H, Ruggieri, L, Turner, M A, Whitaker, M J   +17 more
core   +1 more source

Patient-reported outcome claims in European and United States orphan drug approvals

Journal of Market Access & Health Policy, 2018
Purpose: We aimed to evaluate the rate of usage and the kind of patient-reported outcome (PRO) claims in orphan drug approvals from the European Medicines Agency (EMA) dated between 1/1/2012 and 31/12/2016 and to compare them to those from the US Food ...
S. Jarosławski, P. Auquier, B. Borissov, C. Dussart, M. Toumi   +4 more
semanticscholar   +1 more source

Expediting Drug Development in Japan: A PMDA Perspective

Clinical Pharmacology &Therapeutics, EarlyView.
Review time for a new drug in Japan has shortened dramatically since the establishment of the Pharmaceuticals and Medical Devices Agency (PMDA). Nonetheless, Japan faces a new challenge so‐called “Drug Loss”, which means that new drugs approved overseas have not yet been developed in Japan or that development is delayed. In this manuscript, we describe
Yoichi Kohno, Akihiro Ishiguro, Takashi Yasukawa, Naoyuki Yasuda, Daisuke Tanaka, Yasuhiro Araki, Yukio Takahashi, Yoshiaki Uyama, Yasuhiro Fujiwara   +8 more
wiley   +1 more source

Orphan drug: Development trends and strategies

Journal of Pharmacy and Bioallied Sciences, 2010
The growth of pharma industries has slowed in recent years because of various reasons such as patent expiries, generic competition, drying pipelines, and increasingly stringent regulatory guidelines. Many blockbuster drugs will loose their exclusivity in
Aarti Sharma, Abraham Jacob, Manas Tandon, Dushyant Kumar   +3 more
doaj   +1 more source

Rare diseases and orphan drugs [PDF]

British Journal of Clinical Pharmacology, 2006
The word orphan comes from the Greek word orphanos, a child who has lost one parent or both, or an adult who has lost a child. It goes back to the putative Indo-European root ORBH, bereft, as in the Latin word orbus. The obsolete English words orbation and orbity meant orphanhood or childlessness.
openaire   +4 more sources

Exercise limitations in amyloid cardiomyopathy assessed by cardiopulmonary exercise testing—A multicentre study

ESC Heart Failure, Volume 12, Issue 2, Page 1326-1335, April 2025.
Abstract Aims Amyloid cardiomyopathy is caused by the deposition of light chain (AL) or transthyretin amyloid (ATTR) fibrils, that leads to a restrictive cardiomyopathy, often resulting in heart failure (HF) with preserved or reduced ejection fraction.
Robin Willixhofer, Mauro Contini, Michele Emdin, Damiano Magrì, Alice Bonomi, Elisabetta Salvioni, Fabrizio Celeste, Alberico Del Torto, Claudio Passino, Christophe D.J. Capelle, Chiara Arzilli, Emiliano Fiori, Nicolò Capra, Christina Kronberger, Nikita Ermolaev, Andreas Kammerlander, Beatrice Musumeci, Giuseppe Vergaro, Vincenzo Castiglione, René Rettl, Giacomo Tini, Andrea Baggiano, Iacopo Fabiani, Susanna Sciomer, Roza Badr Eslam, Piergiuseppe Agostoni   +25 more
wiley   +1 more source

Phylogenomic analysis reveals extensive phylogenetic mosaicism in the Human GPCR Superfamily [PDF]

, 2007
A novel high throughput phylogenomic analysis (HTP) was applied to the rhodopsin G-protein coupled receptor (GPCR) family. Instances of phylogenetic mosaicism between receptors were found to be frequent, often as instances of correlated mosaicism and ...
Allaby, Robin G., Woodwark, Mathew
core   +2 more sources

Drivers of Orphan Drug Development.

ACS Medicinal Chemistry Letters, 2018
Previously neglected by the pharmaceutical industry, rare diseases and orphan drugs are rapidly becoming mainstream. Both market forces and technology enablers are responsible for this migration and combine to create multiple business approaches.
Ana Mingorance
semanticscholar   +1 more source

Rest‐activity rhythm phenotypes in adults with epilepsy and intellectual disability

Epilepsia Open, EarlyView.
Abstract Objective Rest‐activity rhythms (RARs) are perturbed in many forms of neuropsychiatric illness. In this study, we applied wrist actigraphy to describe RAR perturbations in intellectually disabled adults with epilepsy (“E + ID”), using a cross‐sectional case–control design.
Nandani Adhyapak, Grace E. Cardenas, Mark A. Abboud, Vaishnav Krishnan   +3 more
wiley   +1 more source