Sanfilippo News

October 22, 2020October 22, 2020

Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series ... Read more

October 15, 2020October 15, 2020

Supporters Gear Up for Nov. 16 – World Sanfilippo Awareness Day

Sanfilippo News

On Nov. 16, World Sanfilippo Awareness Day, supporters will bring global attention to the rare neurodegenerative disorder, hopefully sparking conversations globally about the community and its needs. From sharing images ... Read more

October 1, 2020October 1, 2020

Early Diagnosis of Sanfilippo Syndrome Type C Difficult But Key, Report Shows

Sanfilippo News

An accurate and early diagnosis of Sanfilippo syndrome type C can be difficult, as this subtype is quite rare, but important to prevent life-threatening complications and improve life quality, a case ... Read more

September 25, 2020September 25, 2020

Cure Sanfilippo Foundation Aims to Educate on Remote Learning for Special Needs Children

COVID-19 News, Sanfilippo News

A new initiative led by the Cure Sanfilippo Foundation is seeking to provide more insight on the impact of remote learning on children with Sanfilippo syndrome and other developmental disorders. The ... Read more

September 17, 2020September 17, 2020

AllStripes Asking Patients, Families to Share Health Records to Aid Research

Sanfilippo News

Sanfilippo syndrome patients and caregivers are being asked to help speed the development of potential disease treatments by joining AllStripes, and contributing their de-identified medical records to research efforts. The San ... Read more

September 10, 2020September 10, 2020

How Sanfilippo and Like Disorders Affect 5 Senses Lacks Research

Sanfilippo News

Problems with one or more senses are common in people with Sanfilippo syndrome and other mucopolysaccharidoses (MPSs), but there is very little research into how these impairments affect daily living ... Read more

September 3, 2020September 3, 2020

GlycoNet Grants $3.9M to Canadian Researchers to Study Sanfilippo, Other Diseases

Sanfilippo News

The Canadian Glycomics Network (GlycoNet) has granted $3.9 million in funding for research projects aimed at mitigating the healthcare burden in Canada and contributing to a better understanding of diseases, ... Read more

August 27, 2020September 27, 2020

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

Sanfilippo News

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input ... Read more

August 27, 2020August 27, 2020

SLS-005 Given Orphan Drug Designation for Sanfilippo in EU

Sanfilippo News

The European Medicines Agency has granted orphan drug designation to SLS-005 (trehalose) for the treatment of Sanfilippo syndrome. This designation, which follows a positive opinion from the agency’s Committee for ... Read more

August 20, 2020September 20, 2020

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

Sanfilippo News

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The ... Read more

August 20, 2020August 20, 2020

EMA Committee Gives Positive Opinion on Orphan Drug Status for SLS-005

Sanfilippo News

Seelos Therapeutics announced it has received a positive opinion on an orphan drug designation for SLS-005 (trehalose) — its investigational therapy for Sanfilippo syndrome — from the Committee for Orphan Medicinal ... Read more

August 13, 2020September 13, 2020

New Streaming Channel Showcases Rare Disease Films

COVID-19 News, Sanfilippo News

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films ... Read more

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