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August 27, 2020August 27, 2020

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

News, syndicated

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 ... Read more

August 27, 2020August 27, 2020

SLS-005 Given Orphan Drug Designation for Sanfilippo in EU

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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 ... Read more

August 20, 2020August 20, 2020

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

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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. ... Read more

August 20, 2020August 20, 2020

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

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 ... Read more

August 13, 2020August 13, 2020

New Streaming Channel Showcases Rare Disease Films

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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 ... Read more

August 6, 2020August 7, 2020

Stem Cell Therapy May Ease Neurological Symptoms of Sanfilippo Type A, Study Suggests

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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 ... Read more

July 30, 2020July 30, 2020

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

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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 ... Read more

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

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 ... Read more

July 10, 2020July 10, 2020

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

News, syndicated

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

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