Ultragenyx Pharmaceutical is a mid-cap biotech firm with a promising pipeline and an optimistic outlook from analysts. Click ...

Tierney LaSotta was diagnosed with Sanfilippo Syndrome, a rare condition also known as Childhood Dementia because of its ...

Discover Sanfilippo Syndrome a rare genetic disorder affecting childrens development Learn about its symptoms treatment ...

What is Cushing's syndrome? Cushing's syndrome is a hormonal disorder. It’s caused when you have high levels of the hormone cortisol over a long time. Cushing's syndrome is fairly rare. It most often ...

DNLI has had a good run in the past year on positive pipeline updates. A potential approval of its Hunter Syndrome drug should be a boost. However, recent pipeline setbacks warrant caution.

A young football fan is surprised by the sweetest gift ever. A Dunkin’ Donuts in Lansing surprised 4-year-old Clark Willmore ...

Notre Dame and Ohio State will face off in the College Football National Championships. One four-year-old superfan will get ...

We recently published a list of the 12 Biotech Stocks with the Biggest Upside Potential. In this article, we are going to take a look at where Ultragenyx Pharmaceutical, Inc.

Denali Therapeutics showcases its BBB platform at J.P. Morgan Conference, highlighting DNL310's Hunter syndrome progress and plans for commercialization.

an antisense oligonucleotide for the treatment of Angelman syndrome; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare ...

Clark Willmore, a 4-year-old from Michigan, was diagnosed with Sanfilippo syndrome, a rare genetic disorder affecting the brain and spinal cord, just days before attending his first Ohio State ...

recently diagnosed with Sanfilippo syndrome, a rare genetic disorder affecting the brain and spinal cord. Clark is a big Buckeye fan, and through the work of fans, he was able to attend his first ...