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January 17, 2019January 17, 2019

NAGLU Enzyme Replacement Therapy Fails to Improve Clinical Outcomes, Trial Results Show

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Restoring the activity of the alpha-N-acetylglucosaminidase (NAGLU) enzyme using enzyme replacement therapy (ERT) is well-tolerated by patients with Mucopolysaccharidosis IIIB (MPS IIIB), but fails to improve clinical outcomes, a ... Read more

December 20, 2018December 20, 2018

Abeona Will Include Early-stage MPS IIIA Patients in ABO-102 Gene Therapy Trial

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Abeona Therapeutics is planning to expand its ongoing ABO-102 gene therapy Phase 1/2 trial to include patients with Sanfilippo syndrome type A (MPS IIIA) at earlier stages of the ... Read more

December 20, 2018December 20, 2018

Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish

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Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to ... Read more

December 13, 2018December 13, 2018

New Stem Cell Lines Derived From MPSIIIB Patient May Help Improve Preclinical Research

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Two new experimental stem cell lines derived from a patient with mucopolysaccharydosis IIIB (MPSIIIB), also known as Sanfilippo syndrome type B, were developed by Spanish researchers. These stem cells ... Read more

December 6, 2018December 6, 2018

Family Known as Face of Sanfilippo Syndrome Keeps Working for a Cure

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They had a son they adored, and now Cara and Glenn O’Neill longed for a girl. After a couple of miscarriages, they were overjoyed at the birth of Eliza. ... Read more

November 29, 2018November 29, 2018

Phoenix Nest Gains Rights to Potential Gene Therapy for Sanfilippo Syndrome Type C

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Phoenix Nest has acquired the exclusive rights from the University of Manchester for the development of a gene therapy to treat Sanfilippo syndrome type C. The company is planning to apply for ... Read more

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

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