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 ...
KNOXVILLE, Tenn. — Bryan and Blair Begbie said all they want for Rowan — their youngest daughter — is the chance to be a kid. However, the Food and Drug Administration issued what is called a Complete ...
As parents watch their kids transition from infancy into childhood, it’s common to look for signs of progressing development. Are they sleeping through the night? Can they remember new words and use ...
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 ...
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 ...
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, ...
As a pediatrician, I’ve dedicated my career to advocating for children’s health. But when my daughter, Eliza, was diagnosed with Sanfilippo syndrome, a fatal childhood neurological disease, I realized ...
NOVATO, Calif., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (RARE) today announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration ...
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