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November 15, 2018November 15, 2018

Study Gathers Parents’ Experiences of Caring for Children with Mucopolysaccharidosis

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Caring for a child with mucopolysaccharoidosis (MPS), including Sanfilippo syndrome, can affect all dimensions of a family’s life, yet parents gradually find ways to cope with the difficulties brought ... Read more

November 8, 2018November 8, 2018

Shire’s Investigational ERT Shows Limited Benefits for Sanfilippo Type A Children in Phase 2b Trial

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Administration of an engineered version of the heparan-N-sulfatase (HNS) enzyme into the cerebrospinal fluid directly through the spinal canal failed to improve neurocognitive activity in infants with early-stage Sanfilippo syndrome ... Read more

November 1, 2018November 1, 2018

Combined Behavioral, Pharmacological Therapy Seems to be Best in Treating MPS Disorders

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Combined behavioral and pharmacological therapy seems to be the best course of action in treating behavioral and sleep problems in children with mucopolysaccharidosis (MPS) disorders, a review study has ... Read more

October 30, 2018October 29, 2018

#NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach

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With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Creditand ... Read more

October 25, 2018October 25, 2018

Sanfilippo Type A Causes Toxic Buildup of Several Molecules in Various Brain Regions, Mouse Study Shows

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Sanfilippo syndrome type A leads to toxic accumulation of several molecules in nerve cell axons — the structures responsible for conducting messages — of different brain regions, which correlates with ... Read more

October 23, 2018October 23, 2018

Experts Release Recommendations for Future Sanfilippo Syndrome Trials

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A group of experts has discussed and released a series of recommendations for future trials’ design in Sanfilippo syndrome. The study, “Recommendations on clinical trial design for treatment of ... Read more

October 18, 2018October 18, 2018

Sarepta and Lysogene Sign Licensing Deal for LYS-SAF302, Potential Gene Therapy for Sanfilippo Type A

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Sarepta Therapeutics has signed a licensing agreement with Lysogene for the development of investigational gene therapy LYS-SAF302 to treat mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome ... Read more

October 11, 2018October 11, 2018

Lower Temperatures Improve NAGLU Activity in MPSIIIB Patients’ Cells, Study Shows

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Culturing skin cells from patients with Mucopolysaccharidosis IIIB (MPS IIIB) at lower temperatures promotes the production of the active form of the alpha-N-acetylglucosaminidase (NAGLU) enzyme, researchers have found. These results may ... Read more

October 9, 2018October 9, 2018

Advocacy Groups, Doctors Question Rising Prices of Rare Disease Treatments

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Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced to seven years in federal prison for securities and wire fraud while heading San Diego-based ... Read more

October 4, 2018October 4, 2018

Later Stages of Sanfilippo Syndrome Type A Associated with Severe Decline in Communication, Daily Living, Socialization and Motor Skills, Study Reveals

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Patients with Sanfilippo syndrome type A function below a two-year-old level in terms of communication, daily living skills and socialization, according to a small follow-up study of the later stages ... Read more

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