CIRM Awards $12 Million Grant for Clinical Study of Gene Therapy for Cystinosis Patients
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CIRM Awards $12 Million Grant for Clinical Study of Gene Therapy for Cystinosis Patients

According to a press release from the California Institute for Regenerative Medicine (CIRM), the Institute's governing body has approved a grant of nearly $12 million to University of California, San…

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Diagnostic Timeline for Lysosomal Storage Disorders like Fabry Disease Significantly Reduced in South Africa

The Problem Lysosomal storage disorders (LSDs) affect approximately one out of every 7,000 people. There are around 50 different types of LSDs. These include Fabry disease, Batten disease, Gaucher disease,…

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Kidney Disease Drug May Have Future Treating Mitochondrial Disorders
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Kidney Disease Drug May Have Future Treating Mitochondrial Disorders

A publication at Science Daily recently announced that findings from Children's Hospital of Philadelphia suggested that cysteamine bitartrate, already used for nephropathic cystinosis (a rare kidney disease), might potentially benefit patients…

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Is There a Genetic Condition in Your Family?
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Is There a Genetic Condition in Your Family?

Is there a genetic condition in your family? Are you concerned about the potential for passing this on to your children one day? Do you know if you are a…

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Dad Plans to Promote Awareness About Cystinosis, His Daughter’s Rare Disease as Father’s Day Approaches
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Dad Plans to Promote Awareness About Cystinosis, His Daughter’s Rare Disease as Father’s Day Approaches

According to a story from CBS News, Clay Emerson, from New Jersey, is hoping to use Father's Day in order to raise awareness about his three year old daughter's rare…

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Celebrate a Holiday your Kidney will appreciate: World Kidney Month!
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Celebrate a Holiday your Kidney will appreciate: World Kidney Month!

Last week was World Kidney Day! And in fact, the whole month of March is kidney month - so there's no excuse to brush up on some key kidney facts…

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