A Father Shares the Emotional Reality of Raising a Child with Sanfilippo Syndrome
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A Father Shares the Emotional Reality of Raising a Child with Sanfilippo Syndrome

Terrence Merrill shares the challenging emotional realities of raising a child with Sanfilippo syndrome. You can read more background about the Merrill's battle against this rare disease in our article here.…

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A Gene Therapy Being Developed to Treat Batten Disease Has Been Granted Orphan Drug Status in Europe
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A Gene Therapy Being Developed to Treat Batten Disease Has Been Granted Orphan Drug Status in Europe

A gene therapy called ABO-202 that is being developed to treat Batten disease has just been granted Orphan Drug Designation by the European Medicines Agency (EMA), reports BioPortfolio. This follows…

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How a Local Community Supports a Child with Duchenne Muscular Dystrophy
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How a Local Community Supports a Child with Duchenne Muscular Dystrophy

The phrase “it takes a village” gets tossed around a lot. But in Codicote, that’s what’s happening. Fundraising events and challenges are being held left and right. All of them…

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The FDA Approves a Treatment for Patients with Von Willebrand Disease Undergoing Surgery
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The FDA Approves a Treatment for Patients with Von Willebrand Disease Undergoing Surgery

The US Food and Drug Administration (FDA) has just approved the drug Vonvendi for use in adult patients with von Willebrand disease who are undergoing surgery. This is an extension…

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A Treatment for Muscular Dystrophy Also Works for a Rare Heart Condition
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A Treatment for Muscular Dystrophy Also Works for a Rare Heart Condition

Researchers at the University of Hong Kong have discovered that a drug used to treat muscular dystrophy also works on a hereditary heart disease called Lamin A/C-related dilated cardiomyopathy. The…

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Research into a Genetic Mutation Has Led to the Discovery of a Rare Disease
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Research into a Genetic Mutation Has Led to the Discovery of a Rare Disease

A group of researchers led by Professor Gholson Lyon has discovered a genetic mutation that is linked to certain traits, reports Medical Xpress. The method used to discover it was…

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