Results 211 to 220 of about 68,610 (290)

How to alleviate the shortage: a scoping review of prevention and management of global drug shortages. [PDF]

BMC Public Health
Yi Q, Yang N, Zeng L, Huang L, Zou K, Chen J, Luo J, Zhang M, Diao S, Yu Q, Zhu Y, Han L, Zhang L.   +12 more
europepmc   +1 more source

Supply and Demand in the Mathematics of Rare Disease Drug Development: Why Choosing the Right Model Is Crucial

Clinical and Translational Science, Volume 19, Issue 5, May 2026.
ABSTRACT Clinical trials for rare diseases face a fundamental mathematical challenge that conventional randomized controlled trial (RCT) designs cannot overcome. With approximately 95% of the estimated 10,000–16,000 rare diseases lacking approved therapies, and drug development programs failing at rates exceeding 75% in non‐oncology indications, the ...
Marshall L. Summar, Janet Woodcock
wiley   +1 more source

Strategic Drivers Behind Early Withdrawal of Orphan Designations in the EU: A Retrospective Analysis (2000-2024). [PDF]

Ther Innov Regul Sci
Bouwman L, Castanheira M, Siotis G.
europepmc   +1 more source

Elranatamab: Mechanism of Action, Clinical, and Translational Science

Clinical and Translational Science, Volume 19, Issue 5, May 2026.
ABSTRACT Elranatamab (ELREXFIO) is a humanized bispecific antibody approved for patients with relapsed/refractory multiple myeloma (RRMM). Elranatamab engages CD3 on T cells and B‐cell maturation antigen (BCMA) on myeloma cells to induce T cell–mediated myeloma cell cytolysis.
Mohamed Elmeliegy, Andrea Viqueira, Erik Vandendries, Pooneh Soltantabar, Hoi‐Kei Lon, Kamrine Poels, Blerta Shtylla, Donald Irby, Kristin Bompiani‐Myers, Thomas O'Brien, Jennifer Hibma   +10 more
wiley   +1 more source

Cannabinoid Therapies in Less-Common Disorders: Clinical Evidence and Formulation Strategies. [PDF]

Diseases
Afonso S, Gonçalves J, Brinca AT, Rosendo LM, Rosado T, Duarte AP, Gallardo E.   +6 more
europepmc   +1 more source

Efficacy and Safety of Prophylaxis With a Plasma‐Derived von Willebrand Factor/Factor VIII Concentrate (Wilate) in Patients With Type 3 von Willebrand Disease—A WIL‐31 Study Sub‐Analysis

European Journal of Haematology, Volume 116, Issue 5, Page 674-681, May 2026.
ABSTRACT Objectives The WIL‐31 study demonstrated efficacy and safety of prophylaxis with the plasma‐derived von Willebrand factor/factor VIII concentrate wilate in von Willebrand disease (VWD) of all types and was the only prospective study with an on‐demand run‐in study as an intra‐individual comparator.
Claudia Djambas Khayat, Leonid Dubey, Adlette Inati, Toshko Lissitchkov, Dzmitry Novik, Elina Peteva, Robert F. Sidonio Jr, Ali T. Taher, Kateryna V. Vilchevska, Vladimir Vdovin, Ana Boban   +10 more
wiley   +1 more source

The Simultaneous Prevention of Multiple Diseases: A "One Ring to Rule Them All" Framework for Redox-Driven Health and Longevity. [PDF]

Nutrients
Silverstein HR, Rizvanov AA, Haines DD, Mahmoud FF, Rose SC, Solovyeva VV, Kitaeva KV, Tosaki A.   +7 more
europepmc   +1 more source

Novel Ligands for the Orphan Receptor GPR151 Modulate Morphine Action

Genes to Cells, Volume 31, Issue 3, May 2026.
The new GPR151 ligands altered the analgesic effects of morphine. ABSTRACT GPR151 is an orphan G‐protein‐coupled receptor expressed on specific neurons. Its expression has been reported to be upregulated in the dorsal root ganglia in mouse nerve injury models, suggesting a role in pain transmission.
Hijiri Yoshida, Takashi Suto, Hiroyuki Hirano, Hiroyuki Osada, Kazuto Nunomura, Bangzhong Lin, Shigeru Saito, Shigeki Takeda   +7 more
wiley   +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

In Vitro Metabolism and Analytical Characterization of SLU‐PP‐332 and SLU‐PP‐915: Novel Pan‐ERR Agonists With Doping Potential

Rapid Communications in Mass Spectrometry, Volume 40, Issue 8, 30 April 2026.
ABSTRACT Rationale Estrogen‐related receptor (ERR) agonists such as the drug candidates SLU‐PP‐332 and SLU‐PP‐915 are currently being investigated as exercise mimetics, given their ability to trigger human physiological processes similar to those initiated by actual physical activity.
Tristan Möller, Oliver Krug, Mario Thevis   +2 more
wiley   +1 more source