Study Data Shows Promise in LYS-SAF302 for Sanfilippo Syndrome Type A

Both the 3rd Annual Gene Therapy for Neurological Disorders meeting and the ADVANCE 2022 Sanfilippo Community Conference were held in July 2022. During both meetings, researchers presented new data from…

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The Burkes Are Racing the Clock to Help Save the Lives of Their Three Daughters With Sanfilippo Syndrome

WCVB NewsCenter 5 recently published a news item featuring Nancy and Paul Burke and their three daughters. The Burke family has been living in the shadows of the deadly Sanfilippo…

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Company Raises $14 Million Towards Sanfilippo Syndrome and Other Rare Disease Research
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Company Raises $14 Million Towards Sanfilippo Syndrome and Other Rare Disease Research

According to a story from Sanfilippo News, the healthcare tech company RDMD has recently announced that it has raised $14 million in Series A financing that will be dedicated towards…

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Free Genetic Testing for Lysosomal Storage Diseases Provided by Invitae, Axovant
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Free Genetic Testing for Lysosomal Storage Diseases Provided by Invitae, Axovant

  According to Batten Disease News, Invitae and Axovant Gene Therapies partnered up to offer free genetic testing in North America. This will focus on children who are thought to…

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RDMD Gets $14 Million in Financing, Partners with UCB to Research Progressive Supranuclear Palsy
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RDMD Gets $14 Million in Financing, Partners with UCB to Research Progressive Supranuclear Palsy

  According to BioSpace, healthcare technology company RDMD has raised $14 million in Series A financing, and developed a partnership with UCB. In addition to growing their platform to discuss…

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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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Can a Brain Implant Treat Dementia Caused by Sanfilippo Syndrome? A Boy’s Story Offers Hope
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Can a Brain Implant Treat Dementia Caused by Sanfilippo Syndrome? A Boy’s Story Offers Hope

According to a story from metro.co.uk, Harley Bond was three years old when he was first diagnosed with Sanfilippo syndrome type B, a rare progressive genetic disorder. Now five years…

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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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Bioscience Companies in Ohio Take Aim at Rare Diseases
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Bioscience Companies in Ohio Take Aim at Rare Diseases

According to a story from BioPortfolio, Rare Disease Day, which took place on February 28th, 2019, serves as a time of reflection on the progress that has been made in…

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New Collaborative Effort Will Test a Potential Treatment for Sanfilippo Syndrome
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New Collaborative Effort Will Test a Potential Treatment for Sanfilippo Syndrome

According to a story from Market Screener the biotechnology company Bioblast Pharma has announced a new partnership with Team Sanfilippo a nonprofit foundation which is committed to medical research related…

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Licensing Agreement Struck for an Experimental Sanfilippo Syndrome Drug
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Licensing Agreement Struck for an Experimental Sanfilippo Syndrome Drug

According to a story from publicnow.com, the drug development company Sarepta Therapeutics recently announced that is has completed negotiations for a licensing agreement with Lysogene, a biopharmaceutical company. This agreement…

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