Interim Gene Therapy Trial Data for Two Different Forms of Sanfilippo Syndrome Holds Promise
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Interim Gene Therapy Trial Data for Two Different Forms of Sanfilippo Syndrome Holds Promise

According to a story from gurufocus.com, the biopharmaceutical company Abeona Therapeutics, Inc., recently announced the presentation of positive interim findings from two different phase 1/2 clinical trials. These trials were…

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Will Therapies for Neurological Disorders Bring Forth a New Era for Biotech?
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Will Therapies for Neurological Disorders Bring Forth a New Era for Biotech?

According to a story from BioBuzz, the innovation of a platform for the delivery of gene therapies could help trigger a golden age of development for this new class of…

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Experimental Treatment for Sanfilippo Syndrome Type B Gets Fast Track Designation
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Experimental Treatment for Sanfilippo Syndrome Type B Gets Fast Track Designation

According to a story from finanznachrichten.de, the biopharmaceutical company Abeona Therapeutics, Inc. recently announced that its experimental medical product ABO-101 has earned Fast Track designation from the US Food and…

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After Reviewing Publications About MPS III, Researchers Say More Studies Need to be Carried Out
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After Reviewing Publications About MPS III, Researchers Say More Studies Need to be Carried Out

Researchers have carried out a literature review to investigate MPS III. After reviewing forty-six papers, they concluded that more research needs to be carried out. You can find the original study…

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A Gene Therapy Being Developed for Sanfilippo Syndrome Has Been Granted RMAT status in the US.
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A Gene Therapy Being Developed for Sanfilippo Syndrome Has Been Granted RMAT status in the US.

A gene therapy being developed as a treatment for Sanfilippo syndrome has just been awarded Regenerative Medicine Advanced Therapy (RMAT) status in the US, reports GlobalGenes. Sanfilippo syndrome is a…

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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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