https://philadelphia.cbslocal.com/2019/09/04/newly-approved-drug-being-called-game-changer-for-people-who-suffer-from-hemophilia/
Newly Approved Drug Being Called ‘Game Changer’ For People Who Suffer From Hemophilia
www.HemophiliaTreatmentReport.com
https://philadelphia.cbslocal.com/2019/09/04/newly-approved-drug-being-called-game-changer-for-people-who-suffer-from-hemophilia/
Newly Approved Drug Being Called ‘Game Changer’ For People Who Suffer From Hemophilia
https://www.biopharmadive.com/news/uniqure-stays-a-step-ahead-in-hemophilia-b/562134/
UniQure stays a step ahead in hemophilia B
https://medicalxpress.com/news/2019-08-pediatric-hematologists-hemophilia-patients.html
Pediatric hematologists introduce a novel way to treat pediatric hemophilia A patients
https://www.sciencedirect.com/science/article/pii/S0268960X18300973?via%3Dihub
https://www.reuters.com/article/us-biomarin-pharma-study/biomarin-says-data-shows-hemophilia-gene-therapy-effects-could-wane-idUSKCN1SY2IX
Biomarin to seek approval for hemophilia gene therapy, but durability questions persist
Biomarin to seek approval for hemophilia gene therapy, but durability questions persist
https://www.ncbi.nlm.nih.gov/pubmed/31096314?dopt=Abstract
Gene Therapy: Paving New Roads in the Treatment of Hemophilia.
Semin Thromb Hemost. 2019 May 16;:
Authors: Yamaguti-Hayakawa GG, Ozelo MC
Abstract
Hemophilia is a monogenic disease with robust clinicolaboratory correlations of severity. These attributes coupled with the availability of experimental animal models have made it an attractive model for gene therapy. The road from animal models to human clinical studies has heralded significant successes, but major issues concerning a previous immunity against adeno-associated virus and transgene optimization remain to be fully resolved. Despite significant advances in gene therapy application, many questions remain pertaining to its use in specific populations such as those with factor inhibitors, those with underlying liver disease, and pediatric patients. Here, the authors provide an update on viral vector and transgene improvements, review the results of recently published gene therapy clinical trials for hemophilia, and discuss the main challenges facing investigators in the field.
PMID: 31096314 [PubMed – as supplied by publisher]
https://www.globenewswire.com/news-release/2019/05/02/1815127/0/en/uniQure-Announces-New-Preclinical-Data-in-Hemophilia-A-and-Fabry-Disease-in-Oral-Presentations-at-the-22nd-ASGCT-Annual-Meeting.html
uniQure Announces New Preclinical Data in Hemophilia A and Fabry Disease in Oral Presentations at the 22nd ASGCT Annual Meeting
“proof-of-concept of AMT-180 demonstrated in hemophilia A mouse model”
https://www.businesswire.com/news/home/20190501005328/en/Sigilon-Therapeutics-Delivers-Oral-Presentation-SIG-001-Hemophilia
https://www.businesswire.com/news/home/20190425005202/en/
https://www.biospace.com/article/on-world-hemophilia-day-some-of-the-top-advances/
https://www.eurekalert.org/pub_releases/2019-04/chop-gfv041519.php
https://www.businesswire.com/news/home/20190402005390/en/Sangamo-Pfizer-Announce-Phase-12-Interim-Data
https://apnews.com/Globe%2520Newswire/c4cb1fde71d3ddf1d8a53baa025e5dbe
Catalyst Biosciences Receives Orphan Designation from the European Commission for Marzeptacog Alfa (Activated)
https://www.forbes.com/sites/ikebrannon/2019/03/15/coming-to-grips-with-the-potential-benefits-of-gene-therapy
https://apnews.com/Business%20Wire/77a82c6d3332463b915bf943d8d18314
https://www.medpagetoday.com/recent-developments/hemophilia/78500
http://archive.is/8Qwyq
https://www.nytimes.com/2018/08/13/health/hemophilia-gene-therapy.html [Read more…] about They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong.
http://www.koreabiomed.com/news/articleView.html?idxno=5237
GC starts P1 trials of hemophilia treatment
GC said Thursday that it has administered the first dose of MG1113, a hemophilia treatment, to a patient participating in phase 1 clinical trial for the drug.
The company plans to evaluate the safety of MG1113 in 49 healthy adults and hemophilia patients at two hospitals — Severance Hospital and Korea University Anam Hospital.
Compared to conventional methods of injecting insufficient blood coagulation factors, MG1113 is a therapeutic agent made of antibodies that activate coagulation factors.
http://fortune.com/2019/02/25/roche-gene-therapy-spark/
Roche Bids $4.8 Billion for U.S.-Based Gene Therapy Specialist Spark
https://www.ptcommunity.com/news/20190221/esperoct-approved-treatment-hemophilia-adults-and-children
https://www.biospace.com/article/novo-nordisk-snags-fda-approval-for-hemophilia-a-treatment/
https://www.pharmacytimes.com/news/new-treatment-for-hemophilia-a-receives-fda-approval
http://www.globenewswire.com/news-release/2019/02/19/1737267/0/en/Novo-Nordisk-receives-US-FDA-approval-of-ESPEROCT-turoctocog-alfa-pegol-N8-GP.html
https://www.biopharmadive.com/news/uniqure-update-bolsters-outlook-for-hemophilia-b-gene-therapy/548138/
https://www.marketwatch.com/press-release/uniqure-announces-updated-clinical-data-from-phase-iib-study-of-amt-061-in-patients-with-hemophilia-b-2019-02-08
https://globenewswire.com/news-release/2019/02/06/1711371/0/en/Catalyst-Biosciences-Announces-Preclinical-Proof-of-Concept-Data-of-Gene-Therapy-Candidate-CB-2679d-GT-for-the-Treatment-of-Hemophilia-B.html
https://www.cnbc.com/2018/11/16/biomarins-hemophilia-gene-therapy-offers-patients-a-hope-for-a-cure.html