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January 16, 2020January 16, 2020

SOBI003 Modified Version of Sulfamidase for Sanfilippo Type A Better at Reaching, Persisting in Brain, Rat Study Suggests

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SOBI003, Swedish Orphan Biovitrum AB (Sobi)’s chemically modified version of sulfamidase — the missing enzyme in Sanfilippo syndrome type A — is superior to its original version at reaching and ... Read more

January 9, 2020January 10, 2020

Two New NAGLU-deficient Stem Cell Lines May Help Study Sanfilippo Type B

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Researchers have developed two induced pluripotent stem cell (iPSC) lines with NAGLU mutations — the underlying cause of Sanfilippo syndrome type B — using CRISPR-Cas9 gene editing technology. Since ... Read more

January 2, 2020January 2, 2020

Lysogene’s Gene Therapy Seen to Correct Enzyme Deficiency in Sanfilippo Type A Mouse Model

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Lysogene’s gene therapy candidate LYS-SAF302 corrected enzymatic deficiency, normalized heparan sulfate levels, and reduced brain lesions and neuroinflammation in a mouse model of Sanfilippo syndrome type A. LYS-SAF302’s use ... Read more

December 19, 2019December 19, 2019

Heparan Sulfate Accumulation May Alter Sulfur Metabolism in Sanfilippo Type B, Mouse Study Suggests

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The accumulation of heparan sulfate in Sanfilippo syndrome type B can alter sulfur metabolism in the body, which seems to be sex- and organ-specific, a mouse study suggests. The ... Read more

December 12, 2019December 12, 2019

Gene Therapy Has Potential to Treat Underlying Cause of Sanfilippo, Review Study Finds

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Gene therapy has the potential to effectively target the underlying cause of Sanfilippo syndrome and delay or prevent neurodegeneration, according to a review study. Data from ongoing and future clinical ... Read more

December 11, 2019December 10, 2019

Gene Therapy Takes Center Stage at 2019 NORD Summit

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With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough ... Read more

December 5, 2019December 5, 2019

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

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Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s ... Read more

November 21, 2019November 21, 2019

Milder and Slow-progressing Forms of Sanfilippo Syndrome Arise Differently Later in Life, Study Finds

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Physicians should be aware that milder and slow-progressing forms of Sanfilippo syndrome may not be associated with severe cognitive impairments and may emerge differently later in life, a study ... Read more

November 14, 2019

Rare Disease Film Festival Highlights Patient and Researcher Unity

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Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. ... Read more

October 31, 2019October 31, 2019

Allievex to Continue BioMarin’s Clinical Program Testing Tralesinidase Alfa for Sanfilippo Type B

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Allievex, a new clinical-stage biotechnology company founded by Pappas Capital, will continue the clinical program of BioMarin Pharmaceutical‘s tralesinidase alfa (formerly known as BMN 250) for the treatment of ... Read more

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