Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Técnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.
Treatment of adult spinal muscular atrophy (SMA) patients with Spinraza (nusinersen, by Biogen) seems to be beneficial, despite the challenges associated with its administration, a small study suggests. Patients reported ... Read more
The Japanese Ministry of Health, Labour and Welfare approved Zolgensma (onasemnogene abeparvovec-xioi) for the treatment of spinal muscular atrophy (SMA) patients under the age of two, Novartis announced. The approval includes toddlers ... Read more
Shift Pharmaceuticals said its investigational lead therapy E1v1.11 has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) as a potential treatment option for spinal muscular atrophy (SMA). SMA ... Read more
The amount of a protein in small liquid-filled sacs that cells release could become a biomarker for spinal muscular atrophy, according to a study.
That’s because the levels of the survival motor ... Read more
AveXis presented positive pulmonary data from an interim analysis of its ongoing Phase 1 trial of AVXS-101 for the treatment of spinal muscular atrophy (SMA) type 1. The presentation, by Richard Shell, M.D., ... Read more
University of Missouri researchers have developed a gene replacement therapy for spinal muscular atrophy with respiratory distress type 1 (SMARD1). The therapy is able to cross the protective blood-brain barrier ... Read more
AveXis presented a positive interim analysis of data from its ongoing Phase 1 trial of AVXS-101 for the treatment of spinal muscular atrophy (SMA) type 1. The presentation, by Jerry Mendell, MD, director ... Read more
Ionis Pharmaceuticals recently presented an update of its ongoing open-label Phase 2 clinical study of nusinersen, often called Ionis-SMN, in infants with spinal muscular atrophy (SMA) at the 2016 American Academy of Neurology meeting. ... Read more
Cure SMA, an organization founded in 1984 to fund and invest in research for spinal muscular atrophy (SMA) treatments, also focuses on the day-to-day realities of patients and families, reaching about 4,000 SMA families every ... Read more
Texas Tech University researchers have identified a possible therapeutic target, the enzyme JNK3, for spinal muscular atrophy (SMA) that is independent of the causing genetic mutation. Their research, titled “Genetic ... Read more