Results 141 to 150 of about 22,997 (249)

Advances in orphan drug development for alpha-1 antitrypsin deficiency: a 2025 update from the FDA and EMA. [PDF]

Ther Adv Respir Dis
Höger P, Ries M, Olivares Rivera A, Ersöz H, Buschulte K, Fähndrich S, Kontogianni K, Herth F, Trudzinski FC.   +8 more
europepmc   +1 more source

Genotype‐Dependent Effects of Mechanical Stretch and GATA4‐Targeted Compound 3i‐1262 in Cardiomyopathy Patient–Derived hiPSC‐Cardiomyocytes

Basic &Clinical Pharmacology &Toxicology, Volume 139, Issue 3, September 2026.
ABSTRACT Genetic hypertrophic and dilated cardiomyopathies (HCM and DCM, respectively) are characterised by structural and functional abnormalities that can lead to heart failure. However, current therapies mainly reduce symptoms. Patient‐derived human induced pluripotent stem cell (hiPSC)–derived cardiomyocytes provide a valuable platform to study ...
Saana Pohjavaara, Sini M. Kinnunen, Heikki Ruskoaho, Katriina Aalto‐Setälä, Mika J. Välimäki, Virpi Talman   +5 more
wiley   +1 more source

Advancing hypoparathyroidism treatment: FDA approval of Palopegteriparatide as a promising orphan drug. [PDF]

Ann Med Surg (Lond)
Shahid SR, Shahid K, Ehsan S, Khalid M, Waafira A.   +4 more
europepmc   +1 more source

Mass spectrometry based identification of AMP‐O‐Tris generated by Thermococcus onnurineus Cas10

FEBS Open Bio, Volume 16, Issue 8, Page 1593-1601, August 2026.
Isolated Thermococcus onnurineus Cas10 generates the noncanonical ATP‐derived product AMP‐O‐Tris while in Tris‐containing buffer as identified via mass spectrometry, revealing relaxed nucleophile selectivity under isolated conditions. These findings suggest that multiprotein Csm complex assembly restricts Cas10 reactivity toward canonical cyclic ...
Su‐Jin Lee, Ga Seul Lee, JinHan Kim, Kwang‐Hyun Park, Seong‐Ryung Go, Jeong Hee Moon, Eui‐Jeon Woo   +6 more
wiley   +1 more source

Integrating Equity in Topic Selection for Orphan Medicinal Products: Policy Implications for Health Technology Assessment in Rare Diseases. [PDF]

Appl Health Econ Health Policy
Hosseini F, Mobinizadeh M, Padula WV.
europepmc   +1 more source

Evaluation and Valuation of the Price of Expensive Medicinal Products: Application of the Discounted Cash Flow to Orphan Drugs

International Journal of Rare Diseases & Disorders, 2018
Nuijten Mark, Fugel Hans Joerg, Vis Jan
openaire   +1 more source

From cramming to binge‐watching: Integrating documentary‐based assessment into a pharmacology and toxicology curriculum—a qualitative study

British Journal of Clinical Pharmacology, Volume 92, Issue 8, Page 2474-2486, August 2026.
Abstract Aims In the context of pharmacology and toxicology education, there is a growing shift toward programmatic assessment models that prioritize longitudinal learning, reflection and development of higher‐order cognitive skills. As part of this transition, we are exploring alternative and more meaningful forms of assessment. This qualitative study
Narin Akrawi, Heleen van der Sijs, Floor van Rosse   +2 more
wiley   +1 more source

Targeting Complement Component 1q Ameliorates Diabetic Endothelial Inflammation Via Orphan Nuclear Receptor 4a1-Dependent Suppression of Nuclear Factor κB Signaling. [PDF]

J Am Heart Assoc
Mao A, Shi X, Li Z, Zhang K, Kan H, Geng L, Zhou T, He D.   +7 more
europepmc   +1 more source

Advanced therapy medicinal products (ATMPs) – An analysis of the global regulatory status

British Journal of Clinical Pharmacology, Volume 92, Issue 8, Page 2909-2916, August 2026.
Abstract Background A rapid progress in the development of advanced therapy medicinal products (ATMPs) has taken place over the past decades. The ATMPs hold great potential but face challenges in the clinical development and the regulatory process.
Allan Cramer, Freja Karuna Hemmingsen Sørup, Marie Lund, Tonny Studsgaard Petersen, Kristian Karstoft   +4 more
wiley   +1 more source

Informing Dose for Pediatric Rare Diseases-A Survey of Recent Orphan Drugs Approvals. [PDF]

Clin Transl Sci
Fletcher EP, Kim C, Mei H, AbuAsal B, Samuels S, Madabushi R, Ramamoorthy A.   +6 more
europepmc   +1 more source