Hemophilia
What's New
Last Posted: Apr 22, 2022
- Examining patient and professional perspectives in the UK for gene therapy in haemophilia.
Woollacott Ione et al. Haemophilia : the official journal of the World Federation of Hemophilia 2022 - Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A
S Fong et al, Nature Medicine, April 11, 2022 - Changing paradigms of hemophilia care across larger specialized treatment centers in the European region.
Windyga Jerzy et al. Therapeutic advances in hematology 2022 1320406207221088462 - A preliminary application of a haemophilia value framework to emerging therapies in haemophilia.
Skinner Mark W et al. Haemophilia : the official journal of the World Federation of Hemophilia 2022 28 Suppl 29-18 - Is the world ready for gene therapy?
Garrison Louis P et al. Haemophilia : the official journal of the World Federation of Hemophilia 2022 28 Suppl 25-8 - Precision Public Health in Action: Enhancing models to predict risk of adverse treatment outcomes in people with hemophilia
CJ Bean et al, CDC Blog Post, March 29, 2022 - Prepare the Way for Hemophilia A Gene Therapy
CD Thornburg, NEJM, March 17, 2022 - Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A
MC Ozelo et al, NEJM, March 17, 2022 - Managing Severe Hemophilia A in Children: Pharmacotherapeutic Options.
Regling Katherine et al. Pediatric health, medicine and therapeutics 2022 1327-35 - Disease of the Week: Hemophilia
CDC, March 2022
More
About Rare Diseases PHGKB
Rare Disease PHGKB is an online, continuously updated, searchable database of published scientific literature, CDC and NIH resources, and other information that address the public health impact and translation of genomic and other precision health discoveries into improved health outcomes related to rare diseases...more
Content Summary
- CDC Information (22)
- NIH Information (8)
- COVID-19 (20)
- CDC Publications (12)
- Human Genome Epidemiologic Studies (155)
- Genomic Tests Evidence Synthesis (8)
- Genomic Tests Guidelines (5)
- Tier-Classified Guidelines (3)
- Pathogen Advanced Molecular Detection (2)
- State Public Health Genomics Programs (7)
- Reviews/Commentaries (87)
- Tools/Methods (4)
- Ethical/Legal and Social Issues (ELSI) (5)
Selected Rare Diseases
- Alpha-1 Antitrypsin Deficiency
- Amyotrophic Lateral Sclerosis
- Brugada Syndrome
- Cerebral Palsy
- Cystic Fibrosis
- Duchenne Muscular Dystrophy
- Eclampsia
- Erythema Multiforme
- Familial Mediterranean Fever
- Fragile X Syndrome
- Gaucher Disease
- Glomerulonephritis
- Graves Disease
- Hemophilia
- Huntington Disease
- Microcephaly
- Myasthenia Gravis
- Phenylketonuria
- Retinitis Pigmentosa
- Severe Combined Immunodeficiency
Disclaimer: Articles listed in the Public Health Knowledge Base are selected by the CDC Office of Public Health Genomics to provide current awareness of the literature and news. Inclusion in the update does not necessarily represent the views of the Centers for Disease Control and Prevention nor does it imply endorsement of the article's methods or findings. CDC and DHHS assume no responsibility for the factual accuracy of the items presented. The selection, omission, or content of items does not imply any endorsement or other position taken by CDC or DHHS. Opinion, findings and conclusions expressed by the original authors of items included in the update, or persons quoted therein, are strictly their own and are in no way meant to represent the opinion or views of CDC or DHHS. References to publications, news sources, and non-CDC Websites are provided solely for informational purposes and do not imply endorsement by CDC or DHHS.
- Page last reviewed:Jul 25, 2022
- Page last updated:Sep 30, 2022
- Content source: