X linked News and Research

RSS
Disease-modifying therapy for Alport syndrome still remains an unmet need

Disease-modifying therapy for Alport syndrome still remains an unmet need

Rettsyndrome.org funds research that will benefit boys and girls with MECP2-related disorders

Rettsyndrome.org funds research that will benefit boys and girls with MECP2-related disorders

New drug could alleviate symptoms of rare musculoskeletal condition

New drug could alleviate symptoms of rare musculoskeletal condition

Sussex scientists discover new genetic defect that causes intellectual disability

Sussex scientists discover new genetic defect that causes intellectual disability

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

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

Research unravels possible factor for female bias in lupus

Research unravels possible factor for female bias in lupus

Inherited form of rickets improves more with new injectable medicine than conventional therapy

Inherited form of rickets improves more with new injectable medicine than conventional therapy

Study findings hold promise for children with DMD

Study findings hold promise for children with DMD

New method modifies blood stem cells to reverse genetic mutation that causes IPEX

New method modifies blood stem cells to reverse genetic mutation that causes IPEX

Researchers identify molecule to fight myotubular myopathy

Researchers identify molecule to fight myotubular myopathy

Small populations of normal T cells could modify disease severity in patients with XLP1

Small populations of normal T cells could modify disease severity in patients with XLP1

First U.S. patient treated with innovative gene therapy at Bascom Palmer Eye Institute

First U.S. patient treated with innovative gene therapy at Bascom Palmer Eye Institute

Mimicking Human Brain Development Using Organoids

Mimicking Human Brain Development Using Organoids

Subcutaneous injection of new protein helps treat hemophilia more effectively

Subcutaneous injection of new protein helps treat hemophilia more effectively

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

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

CHOP experts identify new autoimmune disorder along with personalized treatment for young patient

CHOP experts identify new autoimmune disorder along with personalized treatment for young patient

Seeing the Unexpected: Regulatory T Cells and Basophils

Seeing the Unexpected: Regulatory T Cells and Basophils

Researchers discover protein responsible for X chromosome inactivation

Researchers discover protein responsible for X chromosome inactivation

FDA-approved drug for lymphoma and leukemia may help treat common type of brain tumor

FDA-approved drug for lymphoma and leukemia may help treat common type of brain tumor

Research provides new insights about gender differences in airway disease risk

Research provides new insights about gender differences in airway disease risk