Results 71 to 80 of about 32,298 (248)

Developing a Multicriteria Decision Analysis Framework to Establish Differential Cost-Effectiveness Thresholds for Orphan Drugs in Egypt

Bulletin of Faculty of Pharmacy Cairo University
Introduction High pricing of orphan drugs often restricts patient access. As orphan medicines are usually not cost-effective according to traditional thresholds, countries may choose not to consider the cost-effectiveness criterion in pricing and ...
Ahmad Nader Fasseeh, Baher Elezbawy, Nada Korra, Mary GamaI, Ahmad Seyam, Noha Hayek, Nagwan Abdel Rahman, Sohir Abdelhamid, Nader Fasseeh, Amr Shafik, Ahmed Elagamy, Akram Mahmoud, Amal Samir Sedrak, Khaled Elshazly, Mariam Eldebeiky, Monica Talaat, Noura Maher Mohamed, Rania Ahmed Abdelaziz, Remonda Refaat, Shaimaa Akeel, Randa Adel, Samy Khalil, Sherif Abaza, Zoltán Kaló   +23 more
doaj   +1 more source

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

Rare disease patients protection: theoretical discussion and institutional construction of orphan drug designation in China

Orphanet Journal of Rare Diseases
Orphan drugs play a vital role, often described as the “public goods of public goods.” As a result, they demand robust policy support. Among these, the designation of orphan drugs serves as the pivotal and core element of the national incentive mechanism
Xiaopei Zhao, Mengyao Zhang
doaj   +1 more source

Stereoselective Construction of Benzo‐Fused Tetrahydropyrrole Thiazines From Saccharin

European Journal of Organic Chemistry, EarlyView.
Saccharin was used as a template to construct a series of 10a‐substituted, benzo‐fused, pyrrolo thiazinone dioxides. Thereafter, diastereo‐divergent 10‐keto reduction gave either diastereomeric alcohol in a process that did not rely on the substituent at 10a's identity.
Seanán Doyle, Matthew Kiernan, Jordan Smyth, Ajay Mathew, Conor J. Crawford, Julia Bruno‐Colmenarez, Patrick J. Guiry, Aisha Khalifa, Paul Evans   +8 more
wiley   +1 more source

Policy Networks and Policy Entrepreneurship in the EU: Explaining Structural Policy Change in Pharmaceutical Innovation Incentives and Health Technology Assessment

European Policy Analysis, EarlyView.
ABSTRACT Policy process research has excelled in explaining structural policy change within national settings, but extensions and applications to the EU level have long proven challenging for scholars. Given that the EU is currently experiencing its longest period of Treaty stability since the 1980s—having evolved into a sui generis political system ...
Vassilis Karokis‐Mavrikos
wiley   +1 more source

Effectiveness and tolerability of fenfluramine in pediatric and adult patients with developmental and epileptic encephalopathies: A multicenter, retrospective, real‐world clinical‐practice study

Epilepsia, EarlyView.
Abstract Objective Developmental and epileptic encephalopathies (DEEs) are characterized by drug‐resistant seizures and developmental slowing/regression. We examined the efficacy and tolerability of fenfluramine (FFA) in pediatric and adult patients with Lennox–Gastaut syndrome (LGS), Dravet syndrome (DS), and other DEEs.
Vicente Villanueva, Elena González‐Alguacil, Patricia Smeyers, Sara Hernández, Victor Soto‐Insuga, Alejandro Fernández‐Cabrera, Laura Olivié, Teresa de Santos, Beatriz Parejo, Lucas Iacampo, Belén Ortuño, Javier Aparicio, Montserrat Fuentes, Juan Rodriguez‐Uranga, Paula Martínez‐Agredano, José Carlos Estévez, Angel Aledo‐Serrano, Rocio Martin‐Alvarez, David Conejo, Belen Baena, Blanca Mercedes‐Álvarez, Yolanda López, Alba Sierra‐Marcos, Rocio Trincado‐Lamuño, Mouna Ennazeh, Jesús González de la Aleja, Julia Renau, Maria Dolores Castro‐Vilanova, Fátima Romero‐Aguilera, José M. Serratosa   +29 more
wiley   +1 more source

Progress report on new epilepsy treatments: A summary of the Eighteenth Eilat Conference on New Antiepileptic Drugs and Devices (EILAT XVIII). II. Treatments in more advanced clinical development

Epilepsia, EarlyView.
Abstract This article summarizes data for 13 investigational treatments for which at least preliminary seizure outcome data in patients with epilepsy were reported at the Eighteenth Eilat Conference on New Antiepileptic Drugs and Devices held in Madrid, Spain, on May 3–6, 2026.
Meir Bialer, Cecilie Johannessen Landmark, Matthias J. Koepp, Emilio Perucca, Piero Perucca, Torbjörn Tomson, H. Steve White, Elaine Wirrell   +7 more
wiley   +1 more source

Progress report on new epilepsy treatments: A summary of the Eighteenth Eilat Conference on New Antiepileptic Drugs and Devices (EILAT XVIII). I. Treatments in preclinical and early clinical development

Epilepsia, EarlyView.
Abstract Over the last 34 years, the Eilat Conference on New Antiepileptic Drugs and Devices has provided an interactive forum for stakeholders to discuss investigational and recently licensed treatments for seizures and epilepsy. The Eighteenth Eilat Conference on New Antiepileptic Drugs and Devices (EILAT XVIII) took place in Madrid, Spain, on May 3 ...
Meir Bialer, Cecilie Johannessen Landmark, Matthias J. Koepp, Emilio Perucca, Piero Perucca, Torbjörn Tomson, H. Steve White, Elaine Wirrell   +7 more
wiley   +1 more source

A Phase I study of the pharmacokinetics (PK), bioavailability, dose proportionality, safety, and tolerability of topiramate injection compared to Oral topiramate in healthy adult participants

Epilepsia, EarlyView.
Abstract This Phase I study evaluated the safety, tolerability, and pharmacokinetics of a 30‐min topiramate injection (intravenous [IV] TPM) infusion compared with oral topiramate (oral TPM) in healthy adult participants. In this randomized, open‐label, dose escalation, crossover study with 37 healthy participants received single doses of 50, 100, or ...
Adeboye O. Bamgboye, James C. Cloyd, Bernard D. King, Lisa D. Coles   +3 more
wiley   +1 more source

Orphan Drug Approval in Canada, 1999-2022: A Cross-sectional Study [PDF]

International Journal of Health Policy and Management
The number of drugs for orphan indications has been increasing significantly in Canada and the federal government recently announced an investment of $1.5 billion dollars over 3 years primarily directed at helping to fund the cost of these drugs.
Joel Lexchin
doaj   +1 more source