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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Family Hopes to Raise Sanfilippo Syndrome Awareness through #ChaseTheSigns Campaign
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Family Hopes to Raise Sanfilippo Syndrome Awareness through #ChaseTheSigns Campaign

When Sophia Scott was growing up, her parents noticed that she was displaying a variety of different potentially concerning traits. They began undergoing some tests. Eventually, just one day before…

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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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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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Patient Worthy is Excited to Announce a New Partnership with The B.L.A.I.R. Connection!

A New Partnership Patient Worthy is honored and excited to announce our new partnership with The B.L.A.I.R. Connection. The B.L.A.I.R. Connection was founded by Grey Chapin who knows firsthand what…

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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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New Drug For Mucopolysaccharidosis Gets FDA Approval For Study
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New Drug For Mucopolysaccharidosis Gets FDA Approval For Study

The FDA approved a human study to be conducted by Swedish Orphan Biovitrum AB to treating mucopolysaccharidosis type lllA patients with a new drug called SOB1003, reported European Pharmaceutical Review. Not only…

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Is This What the MPS Research Community Been Missing?
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Is This What the MPS Research Community Been Missing?

Recent findings from research done at the University of Pennslyvania indicate a new biomarker associated with a group of rare metabolic diseases called mucopolysaccharidoses (MPS). A biomarker is a measurable…

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We’re On the Way to Treating an Untreatable Condition
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We’re On the Way to Treating an Untreatable Condition

Lysogene, a biopharmaceutical company, announced at the end of May that enrollment in SAMOS (Sanfilippo A Multinational Observational Study) was completed. This is extremely exciting news for Sanfilippo syndrome (MPS…

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