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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

October 24, 2019October 24, 2019

Growth Problems in Sanfilippo Children Milder Than Those Seen in Other MPS Disorders, Study Suggests

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Growth impairment among mucopolysaccharidosis (MPS) disorders is milder in people with MPS III (Sanfilippo syndrome) and most profound in those with MPS IVA (Morquio A syndrome), a study in ... Read more

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

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