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October 3, 2019October 3, 2019

Sanfilippo Type A Changes Metabolism of Sugar and Fat Molecules in Brain Regions, Mouse Study Shows

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Sanfilippo syndrome type A leads to changes in the metabolism of gangliosides (sugar-fat molecules) and sphingolipids (fat molecules) in different brain regions, and at different stages of the disease, according ... Read more

September 27, 2019September 26, 2019

NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC

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Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board ... Read more

September 26, 2019September 26, 2019

Sanfilippo Should Be Considered in Landau Kleffner Syndrome Diagnoses, Case Report Suggests

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Screening for metabolic disorders, such as Sanfilippo syndrome, should be considered in patients initially diagnosed with Landau Kleffner syndrome, according to a recent case study. The report, “An Uncommon ... Read more

September 19, 2019September 19, 2019

Lysogene Announces Goals for Its MPSIIIA Clinical Program

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After a pivotal start in 2019 to its clinical program for Sanfilippo syndrome type A, Lysogene has announced its new goals for developing a treatment for the disease, also known ... Read more

September 5, 2019September 5, 2019

Alpha-Synuclein Buildup Doesn’t Weigh on Early Stages of Sanfilippo Type A, Mouse Study Suggests

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Accumulation of the protein alpha-synuclein in the brain — a hallmark of neurodegenerative diseases like Parkinson’s — does not factor in the early disease course of Sanfilippo syndrome type A, ... Read more

September 2, 2019August 30, 2019

‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases

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Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest. Two entries in November’s “Disorder: ... Read more

August 29, 2019August 29, 2019

Seelos Therapeutics to Begin Phase 2b/3 Trial of Potential Sanfilippo Therapy Trehalose

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Seelos Therapeutics‘ investigational new drug application for SLS-005 (trehalose) has been accepted by the U.S. Food and Drug Administration (FDA) for the treatment of Sanfilippo syndrome. The company can ... Read more

August 28, 2019

At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing

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Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such ... Read more

August 19, 2019August 19, 2019

Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases

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Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one of just six such people ... Read more

August 8, 2019August 8, 2019

Gene Therapy ABO-102 Preserves Cognitive Development in Young Children with Sanfilippo Type A, Interim Data Show

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The youngest Sanfilippo syndrome type A children treated with the gene therapy candidate ABO-102 continue to have normal neurocognitive development up to 18 months after treatment in an ongoing ... Read more

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