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RMEI, PIM sponsor medical education monograph on HAE

RMEI, PIM sponsor medical education monograph on HAE

Synageva supports Fourth International Rare Disease Day

Synageva supports Fourth International Rare Disease Day

Intellikine, PKD Foundation to investigate novel therapies for patients with PKD

Intellikine, PKD Foundation to investigate novel therapies for patients with PKD

INF2 gene mutation is a major cause for focal segmental glomerulosclerosis

INF2 gene mutation is a major cause for focal segmental glomerulosclerosis

Researchers report gene therapy strategy that improves Beta Thalassemia in mice model

Researchers report gene therapy strategy that improves Beta Thalassemia in mice model

Low levels of microbe-killing molecules improve survival rate of people with CGD

Low levels of microbe-killing molecules improve survival rate of people with CGD

Research finds alteration in KBTBD13 protein causes nemaline myopathy

Research finds alteration in KBTBD13 protein causes nemaline myopathy

German Genetic Diagnostics Act in need of amendment

German Genetic Diagnostics Act in need of amendment

University of Utah makes progress against SMA with support of Families of Spinal Muscular Atrophy

University of Utah makes progress against SMA with support of Families of Spinal Muscular Atrophy

Researchers make significant headway in fight against SMA

Researchers make significant headway in fight against SMA

WRNp controls key step in pluripotent stem cell differentiation: Study

WRNp controls key step in pluripotent stem cell differentiation: Study

CSL Behring granted authorization to market Berinert for HAE in Italy and Luxembourg

CSL Behring granted authorization to market Berinert for HAE in Italy and Luxembourg

Immunosuppressive drug sirolimus improves kidney health of patients with ADPKD: Study

Immunosuppressive drug sirolimus improves kidney health of patients with ADPKD: Study

Progress in development of gene therapies for inherited blindness caused by rod degeneration

Progress in development of gene therapies for inherited blindness caused by rod degeneration

Hormone mimic Octreotide promising for treatment of polycystic liver disease caused by ADPKD: Study

Hormone mimic Octreotide promising for treatment of polycystic liver disease caused by ADPKD: Study

New gene therapy to restore retinal cone function and day vision

New gene therapy to restore retinal cone function and day vision

First case of treating cystic fibrosis and Crohn's disease with infliximab

First case of treating cystic fibrosis and Crohn's disease with infliximab

ViroPharma initiates Phase 2 clinical study to evaluate Cinryze

ViroPharma initiates Phase 2 clinical study to evaluate Cinryze

LabCorp offers 21-marker genetic analysis for testing parentage/relationship

LabCorp offers 21-marker genetic analysis for testing parentage/relationship

ATS releases new clinical policy statement on CCHS

ATS releases new clinical policy statement on CCHS