hemophilia – media

Phase 3 Gene Therapy Study on Hemophilia B Launched by Pfizer

Pfizer has initiated its new work on a phase 3 study of an investigational gene therapy designed to treat hemophilia B.
- Source: MedEdge
- 1,211
- July 23, 2018
Clinical Trials hemophilia Pfizer
Syncona Once More Proves Commitment to Freeline in Series B Funding Round

Syncona has once again proven its commitment to gene therapy start-up, Freeline Therapeutics with a £85 million investment from the £88 million series B funding round. Freeline’s main focus is on gene therapy for Haemophilia B and Farby disease and this latest cash injection ensures Syncosa’s majority stake in the startup.
- Source: Fierce Biotech
- 647
- June 20, 2018
biotech Company Insight funding hemophilia
Busy Sanofi takes a pass on one of Alnylam’s rare disease drugs, FDA offers ‘breakthrough’ status for PhIII

A couple of months after Sanofi $SNY and Alnylam $ALNY retooled their RNAi collaboration so that the French pharma giant could concentrate on their hemophilia therapy while giving up rights to the biotech’s patisiran, the two partners are also going their separate ways on a late-stage drug for rare cases of primary hyperoxaluria type 1.
- Source: Endpts
- 564
- March 14, 2018
hemophilia lumasiran New Approvals RNAi
Gene therapy shows promise against blood-clotting disease

Gene therapy has freed 10 men from nearly all symptoms of hemophilia for a year so far, in a study that fuels hopes that a one-time treatment can give long-lasting help and perhaps even cure the blood disease.
- Source: Usnews
- 517
- December 12, 2017
blood disease gene hemophilia
FDA OKs Genentech’s Gazyva for Untreated Follicular Lymphoma

Twice on Thursday, Genentech, scored two approvals from the U.S. Food and Drug Administration(FDA) – a new medicine for hemophilia and an expansion for its cancer drug Gazyva.
- Source: Biospace
- 593
- November 21, 2017
cancer FDA hemophilia lymphoma New Approvals
FDA approves new treatment Hemlibra (emicizumab-kxwh) to prevent bleeding in certain patients with hemophilia A

The U.S. Food and Drug Administration(FDA) approved Hemlibra (emicizumab-kxwh) to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A who have developed antibodies called Factor VIII (FVIII) inhibitors.
- Source: pharmacist
- 744
- November 20, 2017
FDA Hemlibra hemophilia inhibitor New Approvals

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