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Renaissance in drug development for rare diseases

Once famously described as "orphan diseases, too small to be noticed, too small to be funded" in the Hollywood drama Lorenzo's Oil, rare diseases are getting unprecedented attention today among drug...

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Gene therapy developed by UAB cures pediatric Sanfilippo Syndrome A in animal...

A single session of a gene therapy developed by the Universitat Autònoma de Barcelona (UAB) cures Sanfilippo Syndrome A in animal models. This syndrome is a neurodegenerative disease that affects...

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Study highlights benefits of using multiple injections to treat Sanfilippo...

New insight has been gained into treating an inherited disorder that creates serious neurological and behavioral disabilities in children and usually leads to death in the teen years.

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Abeona Therapeutics obtains FDA Orphan Drug Designations for Sanfilippo...

Abeona Therapeutics, a start-up company created around intellectual property licensed from Nationwide Children's Hospital to develop treatments for Sanfilippo Syndrome Types A and B, has been granted...

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Research finding opens door to potential treatment for MPS IIIB

MPS IIIB is a devastating and currently untreatable disease that causes progressive damage to the brain, leading to profound intellectual disability, dementia and death -- often before reaching adulthood.

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Gene therapy is key to addressing Sanfilippo Syndrome, say Ohio scientists...

Gene therapy is the delivery of DNA into a patient's cells to replace faulty or missing genes—or adds new genes—in an attempt to cure cancer or make changes so the body is better able to fight off...

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New brain cell models created to evaluate therapies for Sanfilippo C syndrome

The Sanfilippo syndrome type C is a severe neurodegenerative disease which appearws during the first years of life and for which there is no treatment yet.

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Study provides new insights into an untreatable form of childhood-onset dementia

Is the eye a window to the brain in Sanfilippo syndrome, an untreatable form of childhood-onset dementia, Australian researchers ask in a new publication.

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A new combination therapy for treating rare, fatal Sanfilippo syndrome in...

A study aiming to develop a new therapeutic technique could bring a revolution in our approach to treating rare, fatal Sanfilippo syndrome, a disorder that affects children as young as 2 years old and...

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Rare Disease Day 2021: Raising awareness of childhood dementia

In the lead-up to Rare Diseases Day – on Sunday February 28 - Associate Professor Kim Hemsley’s studies into childhood dementia are helping to unlock answers that can assist treatment of these largely...

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