A single dose of Abeona Therapeutics’ investigational gene therapy ABO-102 safely preserved normal cognitive development for up to three…
Articles by Marta Figueiredo, PhD
Genetic metabolic diseases, such as Sanfilippo syndrome, may underlie an autism spectrum disorder (ASD) diagnosis, especially when accompanied with additional…
Sanfilippo syndrome’s characteristic buildup — to toxic levels — of the complex sugar molecule heparan…
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of…
A gene therapy delivered through a more effective viral carrier safely and effectively corrected enzymatic deficiency, restored cognitive…
The first wave of COVID-19Â in Europe severely disrupted access to care and raised stress and anxiety in people with…
Parents and relatives of those with Sanfilippo syndrome have a more positive perception of expanded carrier screening (ECS)…
Mucopolysaccharidosis (MPS) type IVA (Morquio A syndrome) is the most common type of MPS in Mexico, followed by…
Researchers have developed an efficient way of screening newborns for eight lysosomal storage diseases — including five mucopolysaccharidosis (MPS) disorders — at…
The U.S. Food and Drug Administration (FDA) has released draft guidelines for the design of clinical trials evaluating investigational therapies…