Results 171 to 180 of about 22,997 (249)

Orphan drug propranolol for infantile hemangioma: ten-year real-world safety data from the FAERS database. [PDF]

Orphanet J Rare Dis
Li X, Zhao X, Yang C, Chen A.
europepmc   +1 more source

Outcome of treatment with trametinib in adults with histiocytic neoplasms in the United Kingdom

British Journal of Haematology, Volume 209, Issue 2, Page 747-754, August 2026.
Trametinib (MEK inhibitor) shows efficacy in refractory and high‐risk adult histiocytic neoplasms. At a median follow‐up of 21.4 months, the clinical response rate was 81% (30/37) and the radiological response was 68% (25/37) (n = 37).
Rodothea Amerikanou, Talha Munir, Kristian Bowles, Christopher McNamara, Nikesh Chavda, Elizabeth Price, Jagdish Adiyodi, Alasdair Coles, Philippa Kelsey, Jeffery Smith, Richard Adams, Hayder Hussein, Rui Zhao, Trung Nguyen, Matthew Collin, Satyen Gohil   +15 more
wiley   +1 more source

Navigating market access after conditional reimbursement: a communication roadmap for disinvesting orphan drugs. [PDF]

Int J Technol Assess Health Care
Abdallah K, Blonda A, Godman B, Huys I, Claes K, Denier Y, Simoens S.   +6 more
europepmc   +1 more source

Integrating Artificial Intelligence Into Drug Discovery From Medicinal Plants: Current Applications and Infrastructural Challenges

Chemical Biology &Drug Design, Volume 108, Issue 2, August 2026.
This review presents a systems‐oriented roadmap for integrating artificial intelligence into medicinal plant drug discovery to overcome persistent bottlenecks like extract complexity. It highlights that advancing toward reproducible therapeutics requires making phytochemical datasets AI‐ready via rigorous harmonization and phyto‐centric foundational ...
Amit Gangwal, Azim Ansari, Mohd Usman Mohd Siddique, Jyotiram Sawale, Suhas Padmane, Raju Wadekar   +5 more
wiley   +1 more source

Correction: Immunometabolic remodeling: new perspectives and strategies for liver transplantation. [PDF]

Front Immunol
Wang L, Sheng X, Shi G, Li Y, Wang D, Wang W, Mi C, Wang S, Du Y, Yang H.   +9 more
europepmc   +1 more source

The Evolving Landscape of Pulmonary Hypertension Treatment: From Vasodilation to Disease Modification

Clinical and Translational Science, Volume 19, Issue 8, August 2026.
ABSTRACT Pulmonary hypertension (PH) is a complex disorder associated with significant morbidity and mortality. PH is characterized by an elevated pulmonary vascular load, leading to right ventricular dysfunction and remodeling, with right heart failure and premature death if left untreated.
Surya Ayalasomayajula, Ednan K. Bajwa, Alexandra G. Cornell, Maria Jose Loureiro, Samuel S. Kim, Julia Richmond DiBello, Eric Sterner, Richard Chen, Islam R. Younis   +8 more
wiley   +1 more source

The imperative for national legislation on rare diseases in China: A policy review and call to action. [PDF]

Intractable Rare Dis Res
Li Y, Liu Z, Huang R, Liu Y.
europepmc   +1 more source

OVERVIEW OF REGULATORY INITIATIVES IN THE EUROPEAN UNION TO STIMULATE RESEARCH AND ACCELERATE ACCESS TO ORPHAN DRUGS AND OTHER HIGH MEDICAL NEED PRODUCTS

Acta Poloniae Pharmaceutica - Drug Research, 2019
Aleksandra Baran-Kooiker, Marcin Czech, Coen Kooiker   +2 more
openaire   +1 more source

Deep‐Learning: An Emerging Tool to Support Model‐Informed Drug Development

Clinical and Translational Science, Volume 19, Issue 8, August 2026.
ABSTRACT Traditional Model‐Informed Drug Development (MIDD) primarily relies on hypothesis‐driven (HD) models based on biological, physiological, and physicochemical principles. While these mechanistic models have been instrumental in drug development and regulatory decision‐making, they may be limited in capturing complex nonlinear relationships. Deep
Roberto Gomeni, Françoise Bressolle‐Gomeni   +1 more
wiley   +1 more source

Addressing challenges along the 'access cascade' for new TB regimens. [PDF]

IJTLD Open
Servello J, de Colombani P, Dall'Olio L, Gargioni G, Raviglione MCB.   +4 more
europepmc   +1 more source