Severe Combined Immunodeficiency News and Research

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Moms of children with rare genetic illness push for wider newborn screening

Moms of children with rare genetic illness push for wider newborn screening

IQWiG assesses benefit of newborn screening for severe combined immunodeficiency

IQWiG assesses benefit of newborn screening for severe combined immunodeficiency

Researchers develop first non-human primate X-SCID models using genome editing techniques

Researchers develop first non-human primate X-SCID models using genome editing techniques

Genome engineering-based methods pave way for new treatment of patients with sickle cell disease

Genome engineering-based methods pave way for new treatment of patients with sickle cell disease

Novel gene therapy can improve symptoms of Bubble Boy disease in young adults

Novel gene therapy can improve symptoms of Bubble Boy disease in young adults

Gene therapy can restore immune systems of children and young adults with SCID-X1

Gene therapy can restore immune systems of children and young adults with SCID-X1

Experimental gene therapy may improve health outcomes for patients with some forms of blood disorders

Experimental gene therapy may improve health outcomes for patients with some forms of blood disorders

Prolong Pharmaceuticals' SANGUINATE granted FDA Orphan Drug Designation for SCD treatment

Prolong Pharmaceuticals' SANGUINATE granted FDA Orphan Drug Designation for SCD treatment

Study: New gene therapy safe, effective for patients with hemophilia B

Study: New gene therapy safe, effective for patients with hemophilia B

Finding could lead to more effective, less invasive treatment for 'bubble boy' disease

Finding could lead to more effective, less invasive treatment for 'bubble boy' disease

UCSD professor wins 2015 Japan Prize

UCSD professor wins 2015 Japan Prize

Gut microbe in patients may tip doctors about SCID

Gut microbe in patients may tip doctors about SCID

Newborn screening for SCID holds promise that affected children can lead healthy lives

Newborn screening for SCID holds promise that affected children can lead healthy lives

Study examines national impact of newborn screening test for SCID

Study examines national impact of newborn screening test for SCID

Newborn screening indicates that incidence of SCID is higher than previously believed

Newborn screening indicates that incidence of SCID is higher than previously believed

Blood-forming stem cell transplantation benefits infants with severe combined immunodeficiency

Blood-forming stem cell transplantation benefits infants with severe combined immunodeficiency

Two pediatricians win March of Dimes/Col. Harland D. Sanders Lifetime Achievement Award in Genetics

Two pediatricians win March of Dimes/Col. Harland D. Sanders Lifetime Achievement Award in Genetics

New form of gene therapy shows promising outcomes for "bubble boy" disease

New form of gene therapy shows promising outcomes for "bubble boy" disease

Genetically engineered cell therapies demonstrate early efficacy, safety in patients with blood disorders

Genetically engineered cell therapies demonstrate early efficacy, safety in patients with blood disorders

EMA expands administration options for Hizentra

EMA expands administration options for Hizentra