Marta Figueiredo, PhD, science writer —

Marta holds a biology degree, a master’s in evolutionary and developmental biology, and a PhD in biomedical sciences from the University of Lisbon, Portugal. She was awarded a research scholarship and a PhD scholarship, and her research focused on the role of several signaling pathways in thymus and parathyroid glands embryonic development. She also previously worked as an assistant professor of an annual one-week embryology course at the University of Lisbon’s Faculty of Medicine.

Articles by Marta Figueiredo

Ultragenyx Acquires ABO-102, Potential Sanfilippo Type A Gene Therapy

Ultragenyx Pharmaceutical has closed an agreement giving it global licensing, manufacturing, and commercialization rights to UX111 (formerly ABO-102), an experimental gene therapy for Sanfilippo syndrome type A being tested in the pivotal Phase 1/2 Transpher A trial. Ultragenyx will assume responsibility for UX111’s clinical program, while the therapy’s developer, …

Europe Grants Orphan Drug Status to JR-441, ERT for Sanfilippo A

The European Commission has granted an orphan drug designation to JR-441, JCR Pharmaceuticals’ brain-penetrating experimental enzyme replacement therapy (ERT) for Sanfilippo syndrome type A, the company has announced. The designation is given to investigative therapies with the potential to be safe and effective for rare, life-threatening, or…

Lysogene Regains Rights to LYS-SAF302 for Sanfilippo Type A

Lysogene is terminating its licensing agreement with Sarepta Therapeutics for LYS-SAF302, its investigational gene therapy for Sanfilippo syndrome type A, also known as mucopolysaccharidosis type IIIA (MPS IIIA). The therapy is being evaluated in children with the condition in a Phase 2/3 clinical trial called AAVance (…

Gene Therapies, Ongoing Sanfilippo Research Part of MPS 2021

Recent advances in treating people with Sanfilippo syndrome and other mucopolysaccharidoses (MPS) were among topics addressed at the 16th International Symposium of MPS and Related Diseases (MPS 2021). The annual event, held virtually July 23–25, was organized by MPS Spain in collaboration with different MPS societies worldwide. The…

Metabolic Disorders May Underlie Some Diagnosed Autism Cases

Genetic metabolic diseases, such as Sanfilippo syndrome, may underlie an autism spectrum disorder (ASD) diagnosis, especially when accompanied with additional symptoms, according to a case series in Turkey. The data suggested that 3.3% of ASD cases may have a metabolism-related genetic cause, which in many cases can be attenuated or…