Researchers Use Gene Therapy To Build Immune Systems For Patients With Bubble-Boy Disease
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Researchers Use Gene Therapy To Build Immune Systems For Patients With Bubble-Boy Disease

Researchers from St. Jude Children's Hospital might have just developed a new gene therapy that can build a healthy, functioning immune system for patients suffering from severe combined immunodeficiency, SCID,…

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The Results of this Study Could Improve Outcomes for Duchenne Muscular Dystrophy
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The Results of this Study Could Improve Outcomes for Duchenne Muscular Dystrophy

Last month, the pharmaceutical company Sarepta Therapeutics announced the long awaited results of a study that measured lung function in patients with Duchenne muscular dystrophy (DMD) after they took eteplirsen,…

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These Rare Disease Treatments Just Became a Lot More Affordable for Australians
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These Rare Disease Treatments Just Became a Lot More Affordable for Australians

The Australian government has announced that almost that new treatments worth $300 million are being added to the Pharmaceutical Benefits Scheme. This program provides subsidized medical treatments to citizens of…

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Medical Professionals Are Starting To Debate Clinical Trial Eligibility Standards
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Medical Professionals Are Starting To Debate Clinical Trial Eligibility Standards

According to an article from the Chicago Tribune, many medical specialists are coming to the realization that the eligibility criteria for clinical trials should be reassessed. The problem is that…

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Treatment Development Getting Close for Patients with Form of Adrenoleukodystrophy
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Treatment Development Getting Close for Patients with Form of Adrenoleukodystrophy

Minoryx Therapeutics is on their way to making strides for patients with adrenomyeloneuropathy, also known as AMN, reports Labiotech. They just started their phase II initiative to test patients for…

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