Results 131 to 140 of about 301,643 (264)
Clinical Pharmacology &Therapeutics, Volume 117, Issue 4, Page 1098-1105, April 2025.In the EU, conditional marketing authorization is a pragmatic tool for early approval of a medicine that fulfills an unmet medical need. In the pharmaceutical legislation, an unmet medical need means that a condition lacks a satisfactory method for diagnosis, prevention, or treatment.
Florian Lasch +2 more
wiley +1 more source
Diverse potential of chimeric antigen receptor‐engineered cell therapy: Beyond cancer
Clinical and Translational Medicine, Volume 15, Issue 4, April 2025.1. In theory, CAR‐engineering therapy can achieve therapeutic effects by targeting cells expressing specific antigens and thereby eliminating or regulating disease‐related cell subpopulations. 2. Although CAR‐engineering therapy is currently only approved for the treatment of cancer, it has also shown potential in non‐tumour diseases. 3.
Lvying Wu, Lingfeng Zhu, Jin Chen
wiley +1 more source
Immune Checkpoint Molecules: A Review on Pathways and Immunotherapy Implications
Immunity, Inflammation and Disease, Volume 13, Issue 4, April 2025.Immune checkpoint molecules are today's leading targets for immunotherapy, especially in the field of cancer. This review summarizes the most important and recent advances in this field and in different types of cancer. ABSTRACT Background Today, treating cancer patients with monoclonal antibodies (mAbs), by targeting immune checkpoints, is one of the ...
Erfan Rezazadeh‐Gavgani +4 more
wiley +1 more source
Nephrotic Syndrome as a Complication of Immune Tolerance in Hemophilia B [PDF]
, 1997 Bruce M. Ewenstein +7 more
openalex +1 more source
Genetic counselors' awareness and attitudes regarding gene therapies
Journal of Genetic Counseling, Volume 34, Issue 2, April 2025.Abstract There are 10 gene therapies (GTs) for hereditary conditions that are currently approved by the Food and Drug Administration (FDA). While prior research demonstrates that the majority of healthcare providers lack knowledge regarding GTs, this has not been explored within the genetic counseling profession.
Chelsey Walsh +4 more
wiley +1 more source
Mutation Rates in Humans. I. Overall and Sex-Specific Rates Obtained from a Population Study of Hemophilia B [PDF]
, 1999 P.M. Green +3 more
openalex +1 more source
Expanded carrier screening for inherited genetic disease using exome and genome sequencing
Journal of Genetic Counseling, Volume 34, Issue 2, April 2025.Abstract The goal of this study was to assess the feasibility of using exome (ES) and genome sequencing (GS) in guiding preconception genetic screening (PCGS) for couples who are planning to conceive by creating a workflow for identifying risk alleles for autosomal recessive (AR) and X‐linked (XL) disorders without the constraints of a predetermined ...
N. Belnap +13 more
wiley +1 more source
Molecular diagnosis of hemophilia A and B. Report of five families from Costa Rica
Revista de Biología Tropical, 2004 Hemophilia Aand B are X-chromosome linked bleeding disorders caused by deficiency of the respective coagulation factor VIII and IX. Affected individuals develop a variable phenotype of hemorrhage caused by a broad range of mutations within the Factor ...
Lizbeth Salazar-Sánchez +8 more
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Laryngoscope Investigative Otolaryngology, Volume 10, Issue 2, April 2025.This article reviews the data for and against the use of aspirin to prevent the growth of vestibular schwannomas. It then proposes a randomized placebo‐controlled double‐blinded study to clarify contradictory retrospective findings. ABSTRACT Objective The primary objective of this study is to review the evidence for aspirin use to prevent the growth of
D. Bradley Welling +4 more
wiley +1 more source