Severe Combined Immunodeficiency News and Research

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Despite failed promises, stem cell advocates again want taxpayers to pony up billions

Despite failed promises, stem cell advocates again want taxpayers to pony up billions

New gene therapy cures babies with fatal 'Bubble Boy' disease

New gene therapy cures babies with fatal 'Bubble Boy' disease

Antibody-based treatment could improve cure rate for blood and immune disorders

Antibody-based treatment could improve cure rate for blood and immune disorders

Leadiant Biosciences announces FDA approval of Revcovi for treating pediatric, adult ADA-SCID patients

Leadiant Biosciences announces FDA approval of Revcovi for treating pediatric, adult ADA-SCID patients

Using novel gene therapy to cure infants born with severe combined immunodeficiency

Using novel gene therapy to cure infants born with severe combined immunodeficiency

Smart Cells launches social campaign to support Rare Disease Day 2018

Smart Cells launches social campaign to support Rare Disease Day 2018

A guide to Severe Combined Immunodeficiency Disease, review released

A guide to Severe Combined Immunodeficiency Disease, review released

New review provides guidance to doctors who may treat infants with ‘bubble boy disease’

New review provides guidance to doctors who may treat infants with ‘bubble boy disease’

St. Jude gene therapy offers hope for infants with 'bubble boy' disease

St. Jude gene therapy offers hope for infants with 'bubble boy' disease

Orchard Therapeutics opens new facility for technical operations in San Francisco Bay area

Orchard Therapeutics opens new facility for technical operations in San Francisco Bay area

Study to determine lowest dose of chemotherapy needed for babies with SCID

Study to determine lowest dose of chemotherapy needed for babies with SCID

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