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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How this Sanfilippo Syndrome Patient Registry is Helping Patients
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How this Sanfilippo Syndrome Patient Registry is Helping Patients

The Connect MPS Patient Registry was created in 2016 to learn more about Sanfilippo syndrome and mucolipidoses (ML). 24 advocacy organizations are a part of this registry, all of which…

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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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One Girl’s Diagnosis Inspired a Foundation for Children with Sanfilippo Syndrome
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One Girl’s Diagnosis Inspired a Foundation for Children with Sanfilippo Syndrome

On the pages of the Abby Grace Foundation website, you will find over twenty beautiful pictures of different children. Most of the children are smiling. One looks into the camera…

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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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