Last Posted: Jun 03, 2021
- Prediction of hemophilia A severity using a small-input machine-learning framework.
Lopes Tiago J S et al. NPJ systems biology and applications 2021 7(1) 22
- Patterns of joint damage in severe haemophilia A treated with prophylaxis.
Goren Rachel et al. Haemophilia : the official journal of the World Federation of Hemophilia 2021
- Optimizing language for effective communication of gene therapy concepts with hemophilia patients: a qualitative study.
Hart Daniel P et al. Orphanet journal of rare diseases 2021 16(1) 189
- Gene therapy may not be as expensive as people think: challenges in assessing the value of single and short-term therapies.
Garrison Louis P et al. Journal of managed care & specialty pharmacy 2021 27(5) 674-681
- Data from CDC’s hemophilia surveillance programs can help prevent or reduce hemophilia-related health problems
CDC, March 2021
- Health care resource utilization and costs among adult patients with hemophilia A on factor VIII prophylaxis: an administrative claims analysis.
Croteau Stacy E et al. Journal of managed care & specialty pharmacy 2021 27(3) 316-326
- Core data set on safety, efficacy, and durability of hemophilia gene therapy for a global registry: Communication from the SSC of the ISTH.
Konkle Barbara et al. Journal of thrombosis and haemostasis : JTH 2020 Nov 18(11) 3074-3077
- Current Clinical Applications of in vivo Gene Therapy with AAVs.
Mendell Jerry R et al. Molecular therapy : the journal of the American Society of Gene Therapy 2020 Dec
- Assessing the current knowledge, attitude and behaviour of adolescents and young adults living with haemophilia.
Mohan Richa et al. Haemophilia : the official journal of the World Federation of Hemophilia 2020 Dec
- Addressing patient education priorities in the era of gene therapy for haemophilia: Towards evidence-informed shared decision-making.
Woollard Laurence et al. Haemophilia : the official journal of the World Federation of Hemophilia 2020 Nov
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
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