Hemophilia A News and Research

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ASGCT seeks to educate public and policy-makers on fundamentals of gene editing

ASGCT seeks to educate public and policy-makers on fundamentals of gene editing

New European study emphasizes need to enhance standard of haemophilia care in real life

New European study emphasizes need to enhance standard of haemophilia care in real life

Two leading non-profit organizations sign strategic affiliation to hasten drug development

Two leading non-profit organizations sign strategic affiliation to hasten drug development

New virtual reality games create better patient-experience for kids during infusions

New virtual reality games create better patient-experience for kids during infusions

Scientists link malfunctioning molecular pathways to specific heart abnormalities in SCA

Scientists link malfunctioning molecular pathways to specific heart abnormalities in SCA

UNC professor identifies research priorities to address VTE in cancer patients

UNC professor identifies research priorities to address VTE in cancer patients

TSRI awarded $20 million for first year of precision medicine initiative cohort program

TSRI awarded $20 million for first year of precision medicine initiative cohort program

Study provides more insight into treatment options for severe hemophilia A

Study provides more insight into treatment options for severe hemophilia A

Scientists use new technique to repair fibrotic liver cells within the organ

Scientists use new technique to repair fibrotic liver cells within the organ

SIPPET study may have implications for treatment of patients with severe hemophilia A

SIPPET study may have implications for treatment of patients with severe hemophilia A

Jumping genes may play crucial role in generation of cancer

Jumping genes may play crucial role in generation of cancer

European Medicines Agency approves first-ever treatment for hereditary factor X deficiency

European Medicines Agency approves first-ever treatment for hereditary factor X deficiency

Research: X and Y DNA swapping may occur more often than previously thought

Research: X and Y DNA swapping may occur more often than previously thought

UC San Diego Health participates in nationwide clinical study on hemophilia B gene therapy

UC San Diego Health participates in nationwide clinical study on hemophilia B gene therapy

Inherited gene after black plague may help treat HIV patients co-infected with hepatitis C

Inherited gene after black plague may help treat HIV patients co-infected with hepatitis C

Idelvion approved for use in children and adults with Hemophilia B

Idelvion approved for use in children and adults with Hemophilia B

Modified protein appears to reverse liver fibrosis, cirrhosis in rats

Modified protein appears to reverse liver fibrosis, cirrhosis in rats

Baxalta seeks FDA approval for ADYNOVATE to treat children with hemophilia A and for use in surgical settings

Baxalta seeks FDA approval for ADYNOVATE to treat children with hemophilia A and for use in surgical settings

FDA clears Sangamo BioSciences' SB-318 IND application for treatment of MPS I

FDA clears Sangamo BioSciences' SB-318 IND application for treatment of MPS I

Gene therapy holds considerable potential for safe, effective treatment of people with factor VII deficiency

Gene therapy holds considerable potential for safe, effective treatment of people with factor VII deficiency