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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

August 1, 2019

Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate

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Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in the United States. No state ... Read more

August 1, 2019

Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card

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Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second in the number of teen births per 100,000 teenage ... Read more

August 1, 2019August 1, 2019

Lysogene Creates Scientific Advisory Board to Advance Gene Therapy in Sanfilippo, Other Disorders

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Lysogene has established a scientific advisory board (SAB) to further explore gene therapy as a treatment for central nervous system (CNS) disorders such as Sanfilippo syndrome. Comprised of global ... Read more

July 25, 2019July 25, 2019

Heart Problems Less Common, Milder in Sanfilippo Than Other MPS Forms, Taiwanese Study Finds

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Heart problems are less common, and milder in severity, in people with Sanfilippo syndrome compared with other forms of mucopolysaccharidosis, a Taiwanese study has found. The cardiac problems identified ... Read more

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