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July 23, 2020July 23, 2020

Potential Sanfilippo Treatment, SLS-005, Advances as Orphan Drug in EU

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The Committee for Orphan Medicinal Products, a branch of the European Medicines Agency (EMA), favors designating SLS-005 (trehalose) an orphan drug as a potential treatment for Sanfilippo syndrome, Seelos Therapeutics, ... Read more

July 16, 2020July 16, 2020

Enrollment, Dosing Underway in Sanfilippo Type A and B Trials of Gene Therapy

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Patients are continuing to be treated in clinical trials testing Abeona Therapeutics‘ investigational gene therapies — ABO-101 for those with Sanfilippo syndrome type B (MPS IIIB), and ABO-102 for Sanfilippo syndrome type A ... Read more

July 10, 2020July 10, 2020

National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US

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The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease ... Read more

July 9, 2020July 9, 2020

Survey Shows Gaps in Acceptance of Genetic Testing With Family Planning

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Parents and relatives of those with Sanfilippo syndrome have a more positive perception of expanded carrier screening (ECS) for pregnancies than the general population, and are more likely to ... Read more

July 2, 2020July 2, 2020

Defects in Autophagy Underlie Eye Symptoms in Sanfilippo Type A, Mouse Study Shows

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An abnormal accumulation of heparan sulfates — a hallmark of Sanfilippo syndrome type A — in the retina causes cell death and induces eye symptoms by impairing a process called autophagy, ... Read more

June 25, 2020June 25, 2020

Anakinra, Known Anti-inflammatory, in Phase 2/3 Trial for Sanfilippo

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An ongoing clinical trial is investigating an approved anti-inflammatory compound anakinra, sold under the brand name Kineret, as a potential treatment for Sanfilippo syndrome. The Phase 2/3 pilot trial (NCT04018755) opened in May ... Read more

June 22, 2020June 22, 2020

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

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The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will ... Read more

June 18, 2020June 18, 2020

Lysogene CEO Karen Aiach, Inspired by Daughter, Determined to Treat Sanfilippo

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Karen Aiach never imagined she’d one day start a drug company dedicated to curing a fatal genetic disease. But that was before 2005, when the French business consultant gave ... Read more

June 11, 2020June 11, 2020

FDA Puts Hold on LYS-SAF302 Gene Therapy Trial for Sanfilippo Type A

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The U.S. Food and Drug Administration (FDA) has issued a hold on Lysogene‘s trial of its gene therapy candidate LYS-SAF302 for Sanfilippo syndrome type A. LYS-SAF302 is a one-time ... Read more

June 10, 2020June 10, 2020

Coalition Will Address Racial Disparities in Rare Disease Communities

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The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for a ... Read more

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