AOL

This Family Advocates For Rare Sanfilippo Syndrome In Hopes That Others Have Better Chances

Not everyone is given equal opportunities in life, but even when faced with the most difficult challenges, it’s not the circumstances that define us, but our outlook. Logan was born in 2007 alongside ...
Yahoo

Teen Battling Sanfilippo Syndrome, aka ‘Childhood Alzheimer's’

Sixteen-year-old Logan Pacl from Washington is living with Sanfilippo syndrome. This rare neurodegenerative disorder, caused by a gene defect, affects the brain and spinal cord. Children lose skills, ...
EurekAlert!

Landmark Nature Medicine study reports promising new treatment reduces suffering in Sanfilippo syndrome

As a neurodegenerative disease characterized by childhood onset dementia, Sanfilippo syndrome causes immense suffering in many ways, including pain, loss of speech, extreme agitation, and distress, ...
Salon

The FDA is failing children with a rare, neglected disease sometimes called “childhood dementia”

The effects of Sanfilippo syndrome in small children are so devastating that the rare disease is often known as "childhood dementia." About one in 70,000 newborns inherit this disorder. The affected ...
News Medical

Anakinra shows promise in reducing symptoms in patients with Sanfilippo syndrome

Sanfilippo syndrome, also known as mucopolysaccharidosis type III (MPS III), is considered an orphan disease, which classifies it for special considerations in drug development and policy. It is a ...
Nasdaq

Ultragenyx Announces New Data For UX111 Gene Therapy In Treating Sanfilippo Syndrome Type A

(RTTNews) - Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company, on Wednesday reported encouraging data from its ongoing clinical program of UX111, a gene therapy for Sanfilippo ...
NBC DFW

Runners lace up to raise money, awareness as Sachse 10-year-old battles rare, fatal genetic syndrome

There are still moments when life feels normal inside of the Sink home. But for John and Katharine Sink, not a day goes by that they don’t think of the diagnosis doctors gave 10-year-old Declan six ...
Nasdaq

Ultragenyx Submits BLA To FDA For UX111 Gene Therapy For Sanfilippo Syndrome Type A

(RTTNews) - Ultragenyx Pharmaceutical Inc. (RARE), announced on Thursday that it has submitted a Biologics License Application to the U.S. FDA for UX111, an AAV gene therapy for Sanfilippo Syndrome ...
WKYC3

College football fans unite to raise thousands for young Ohio State fan with rare disease

COLUMBUS, Ohio — Growing up, Clark Willmore always dealt with a curved back and was "a little clumsy," according to his dad Japeth. His family decided to take Clark to the doctor to learn more about ...

This Biotech Stock Soared 1,400%. Investors Are Optimistic About Its Rare-Disease Drug.

The microcap biotech stock had its best day ever after the FDA granted its rare-disease drug a breakthrough designation.
Business Insider

Abeona Announces $13.85M Grant from Leading Sanfilippo Syndrome Foundations for Clinical Development of MPS III Gene Therapies

Nine global foundations collaborate to grant $13.85 million for the continued advancement of lead Sanfilippo gene therapy programs Company receives infusion of an additional $5 million from exercise ...