News

November 19, 2020November 19, 2020

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

News, syndicated

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about ... Read more

November 12, 2020November 12, 2020

Cause of Death Unknown in Child Participating in LYS-SAF302 Gene Therapy Trial

News

One of the children participating in the Phase 2/3 trial of LYS-SAF302, Lysogene’s gene therapy candidate for Sanfilippo syndrome type A, has died. The exact cause of death is ... Read more

November 6, 2020November 6, 2020

NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits

News, syndicated

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit ... Read more

November 5, 2020November 5, 2020

Hearing Loss Common in Young Patients With MPS Disorders, Study Finds

News

Hearing loss is the most common auditory complaint among young patients with different forms of mucopolysaccharidosis (MPS), including Sanfilippo syndrome, a small study suggests. According to investigators, these findings highlight ... Read more

October 29, 2020October 29, 2020

‘Save Connor’ Video Raising Funds for First-ever Clinical Trial in Sanfilippo Type C

News

An emotional video about a boy with Sanfilippo syndrome and his parents’ fight to save him has raised about $244,000 toward a first-ever clinical trial that could give the youngster ... Read more

October 27, 2020October 28, 2020

UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner

News, syndicated

A photo of a bespectacled young boy, his red baseball cap slightly askew as he enjoys time outside, will be featured on the front cover of an upcoming calendar ... Read more

October 22, 2020October 21, 2020

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

News, syndicated

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 ... Read more

October 15, 2020October 15, 2020

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

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 ... Read more

October 8, 2020October 8, 2020

Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos

News, syndicated

Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare ... Read more

October 1, 2020October 1, 2020

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

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 ... Read more