Results 221 to 230 of about 1,419,402 (298)

Generic orphan drug substitution: a critical analysis of global practices and Saudi Arabia's perspective. [PDF]

Front Pharmacol
Alakeel YS, Rampakakis E, AlRumaih A, AlRuwaisan R, Abushal M, AlDalaan AM, Idrees MM, Alanazi ZD, AlKoait H, Muaadi A, AlAfra MAM, AlShaya SA, AlHomida S.   +12 more
europepmc   +1 more source

Characterization of Adult Patients With Neurometabolic Disorders: A Cross‐Sectional Study at a Tertiary Neurology Center in Sweden

JIMD Reports, Volume 67, Issue 5, September 2026.
ABSTRACT Adult patients with inherited metabolic diseases are often overlooked. Limited data on this population hinder adequate planning of their clinical and social care. In this retrospective, observational, cross‐sectional service evaluation study, we reviewed the electronic medical records of adult patients with inherited neurometabolic diseases ...
Boel Ernerdahl, Ashraf Yahia, Andreas Puschmann   +2 more
wiley   +1 more source

FDA Approval of Orphan Drug Indications for Pediatric Patients, 2011-2023.

JAMA Pediatr
Kakkilaya A, Shahzad M, Bourgeois FT.
europepmc   +1 more source

Recurrent Respiratory Papillomatosis Foundation Position Statement on the Management of Adults With RRP

The Laryngoscope, Volume 136, Issue S4, Page S7-S17, September 2026.
ABSTRACT Objective With regulatory approval of HPV‐specific immunotherapy for recurrent respiratory papillomatosis (RRP) and growing experience with systemic bevacizumab, a management algorithm incorporating these medical treatments is warranted. Data Sources and Methods RRP Foundation (RRPF) Key Opinion Leaders offer a proposed management algorithm ...
Simon R. Best, Aaron D. Friedman, Clark A. Rosen, Robert T. Sataloff, Laura A. Matrka, H. Steven Sims, David E. Rosow, Nabil F. Saba, David G. Lott, Adam M. Klein, Ted Mau, Milan R. Amin, Kathryn A. Wikenheiser‐Brokamp, Scott M. Norberg, Kim McClellan, Geoffrey D. Young, Clint T. Allen, on behalf of the Recurrent Respiratory Papillomatosis Foundation   +17 more
wiley   +1 more source

The Orphan Drug Act at 40: Legislative Triumph and the Challenges of Success. [PDF]

Milbank Q
Saltonstall P, Ross H, Kim PT.
europepmc   +1 more source

Therapeutic Potential of Chickpea‐Derived Bioactives: A Review With Perspective of Different In Vitro, Animal, and Human Studies

Legume Science, Volume 8, Issue 3, September 2026.
ABSTRACT Chickpea (Cicer arietinum L.) is an important dietary legume worldwide and provides micronutrients and phytochemicals, including phenolics, flavonoids, carotenoids, bioactive peptides, dietary fiber, and resistant starch. This narrative review summarizes evidence from in vitro, animal, and human studies on the potential health relevance of ...
Rohama Shahid, Maria Maqsood, Nimrah Ashraf, Farzeen Asghar, Nauman Khalid   +4 more
wiley   +1 more source

Cyclins and Cyclin‐Dependent Kinases: Structure, Biological Functions, and Innovative Targeting Strategies in Cancer

MedComm, Volume 7, Issue 9, September 2026.
This work synthesizes recent insights into the pathological roles of cyclins and cyclin‐dependent kinases (CDKs) across human cancers, highlights state‐of‐the‐art innovative approaches (especially targeted degradation and redistribution of CDK/cyclin proteins) for cancer therapy, and outlines future directions for CDK/cyclin‐related biomedical research.
Suya Zheng, Zhipeng Shen, Yuanfang Wu, Xingze Huang, Zhongyuan Zhang, Jiangyu Liu, Qichun Wei, Wenhao Chen, Ye Chen, Liang Xu   +9 more
wiley   +1 more source

Safety and Efficacy of Vedolizumab for the Patients With Gastrointestinal Acute Graft‐Versus‐Host Disease Refractory to Steroid Treatment After Allogeneic Hematopoietic Stem Cell Transplantation: A Real‐World, Retrospective Multicenter Study (VAST Study)

MedComm, Volume 7, Issue 9, September 2026.
This multicenter, real‐world study with a large sample size (i.e., vedolizumab for aGVHD refractory to steroid treatment, VAST study) was designed to identify the safety and efficacy of vedolizumab treatment in patients with steroid‐refractory gastrointestinal acute graft‐versus‐host disease (SR‐GI‐aGVHD). The overall response rates (ORR) on day 28 and
Leqing Cao, Yi Xia, Song Xue, Jianping Zhang, Jiameng Li, Yuewen Wang, Qi Wen, Ze Tian, Jun Kong, Daoxing Deng, Xiaohui Zhang, Yang Cao, Na Xu, Xiaodong Mo   +13 more
wiley   +1 more source

Oxysterols in Cancer: From Biosynthesis and Pathophysiology to Targeted Therapeutics

MedComm – Oncology, Volume 5, Issue 3, September 2026.
Mechanisms by which oxysterol targeting may enhance immunotherapy efficacy. This figure illustrates potential metabolic interventions that may improve antitumor immunity by modulating oxysterol related pathways. CH25H modulation may reduce 25‐HC accumulation, promote the conversion of cold tumors into hot tumors, and increase T cell infiltration and PD‐
Haili Shang, Yongsheng Li
wiley   +1 more source

Predictors of orphan drug approval

Orphanet Journal of Rare Diseases, 2010
Heemstra Harald
doaj   +1 more source