Sanfilippo News

August 6, 2020August 7, 2020

Stem Cell Therapy May Ease Neurological Symptoms of Sanfilippo Type A

Sanfilippo News

Treatment with blood stem cells genetically engineered to block interleukin-1 beta (IL-1b) signaling may be a promising approach for reducing brain inflammation and cognitive decline in Sanfilippo syndrome type A, ... Read more

July 30, 2020July 30, 2020

Participants Sought for Study to Help Predict Types, Severity of MPS Disorders

Sanfilippo News

The University of Washington and the National MPS Society are seeking  participants for a study to predict the type and severity of MPS (mucopolysaccharidosis) disorders, such as Sanfilippo syndrome (MPS III). Results of ... Read more

July 23, 2020July 23, 2020

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

Sanfilippo News

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, its ... Read more

July 16, 2020July 17, 2020

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

COVID-19 News, Sanfilippo News

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 (MPS ... Read more

July 9, 2020July 9, 2020

Survey Shows Gaps in Acceptance of Genetic Testing With Family Planning

Sanfilippo News

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 make ... Read more

July 2, 2020July 6, 2020

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

Sanfilippo News

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, which ... Read more

June 25, 2020June 26, 2020

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

Sanfilippo News

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 in ... Read more

June 22, 2020July 23, 2020

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

COVID-19 News, Sanfilippo News

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 support ... Read more

June 18, 2020June 18, 2020

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

Sanfilippo News

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 birth ... Read more

June 11, 2020June 11, 2020

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

Sanfilippo News

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 treatment ... Read more

June 10, 2020July 11, 2020

Coalition Will Address Racial Disparities in Rare Disease Communities

COVID-19 News, Sanfilippo News

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 disease ... Read more

June 4, 2020June 4, 2020

Seelos Seeks Natural History Data, Rare Disease and Orphan Drug Designations for SLS-005

Sanfilippo News

Seelos Therapeutics and the U.S. Food and Drug Administration (FDA) are continuing to discuss the use of Sanfilippo syndrome natural history data as a control for a pivotal U.S. study ... Read more

May 28, 2020June 28, 2020

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

COVID-19 News, Sanfilippo News

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies ... Read more

May 28, 2020May 28, 2020

Cure Sanfilippo Grants $380,000 to Type C Gene Therapy Development

Sanfilippo News

Cure Sanfilippo Foundation has granted $380,000 to the New York-based biotechnology company Phoenix Nest to support the development of the first-ever gene therapy to treat children with the rare genetic ... Read more

May 25, 2020June 25, 2020

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Sanfilippo News

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was ... Read more