Posts by: Margarida Azevedo

Margarida Azevedo

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.

June 17, 2020June 17, 2020

CRISPR/Cas9 Gene Editing May Correct Genetic Defect in DEB, Study Suggests

Epidermolysis Bullosa News

A CRISPR/Cas9-based gene editing strategy was able to restore production of type VII collagen protein (C7) in skin cells from a patient with severe dystrophic epidermolysis bullosa (DEB), a study ... Read more

April 22, 2020April 22, 2020

Silica Particles May Be Way of Oral Enzyme Replacement Therapy for PKU

Phenylketonuria News

A carrier system made of tiny silica particles may be used to deliver a therapeutic enzyme directly to the digestive system, a recent study suggests. Such an oral treatment would ... Read more

April 22, 2020April 22, 2020

Oral Dapsone Can Lead to Rapid, Sustained Improvement in Inherited EB, Case Report Shows

Epidermolysis Bullosa News

Oral treatment with dapsone, an antibiotic and anti-inflammatory, may be a valuable therapy for patients with inherited epidermolysis bullosa, according to a case report that showed the therapy resulted in ... Read more

April 14, 2020April 14, 2020

Balstilimab, Potential Cervical Cancer Treatment, Placed on Fast Track

Immuno-Oncology News

The U.S. Food and Drug Administration (FDA) has now given fast track status to Agenus’ balstilimab — both alone and in combination — as an immunotherapy candidate for advanced metastatic cervical cancer. This designation, given ... Read more

April 3, 2020April 3, 2020

Phase 3 Results Suggest Low-Dose Prednisone Lowers Flare Risk in SLE Patients in Remission

Lupus News Today

People with systemic lupus erythematosus (SLE) who have been in remission for at least a year and take low-dose prednisone as maintenance therapy have less risk of a disease relapse ... Read more

April 3, 2020April 3, 2020

Surgical Patch TachoSil May Be Alternative Therapeutic Option for Corneal Healing in NK, Case Report Suggests

Neurotrophic Keratitis News

TachoSil, a spongy collagen patch that promotes healing post-surgery, was successfully used as a first-line treatment in hard-to-treat corneal defects due to neurotrophic keratitis (NK), a report of two clinical ... Read more

March 30, 2020March 30, 2020

Zevalin Improves Survival of Non-Hodgkin’s Lymphoma Patients in Real-world Setting, Study Finds

Lymphoma News Today

Treatment with Zevalin (ibritumomab tiuxetan), which combines an antibody with a radioactive agent, was found to be safe, reduced tumor burden in most patients, and significantly improved survival without disease worsening ... Read more

March 30, 2020March 30, 2020

Common Antibody Abnormalities in Gaucher Not Linked to Disease Severity, Study Finds

Gaucher Disease News

Although common in patients with Gaucher disease, antibody abnormalities are not associated with disease severity, a large French study reports. Still, monitoring these patients is important because they may develop ... Read more

March 25, 2020March 25, 2020

XLH Symptoms Significantly Affect Well-being of Patients, Study Finds

XLH News Today

People with X-linked hypophosphatemia (XLH) frequently experience pain, stiffness, and fatigue, which have a significant impact on their physical functioning, mental health, and daily lives, a study reports. They also ... Read more

March 24, 2020March 24, 2020

PWS-associated Gene Involved in Integration of Vision and Motor Control in the Brain

Prader-Willi News

Altered gene expression underlies impaired function of brain areas regulating vision and motor control, which may be associated with behavioral issues in people with Prader-Willi syndrome (PWS) and other disorders, ... Read more

March 19, 2020March 21, 2020

SERPINE-1 May Help Predict Response to Gastric Sleeve Surgery, Study Suggests

Genetic Obesity News

Levels of a protein called SERPINE-1 and modifications in its gene may help predict the success of gastric sleeve surgery for obese patients seeking to lose weight and maintain their ... Read more

March 19, 2020March 21, 2020

ELEKTRA Trial Testing Soticlestat Completes Early Enrollment of Children With LGS, Dravet

Lennox-Gastaut Syndrome News

The ELEKTRA trial testing the investigational oral therapy soticlestat (OV935/TAK-935) in children with Lennox-Gastaut syndrome (LGS) and Dravet syndrome has completed patient enrollment, Ovid Therapeutics has announced. “We completed enrollment significantly ahead of ... Read more

March 18, 2020March 21, 2020

Omitting Rituxan Dose Does Not Affect AAV Relapse Rates, Analysis Suggests

ANCA Vasculitis News

Omitting one dose of Rituxan (rituximab) in a previously tested, tailored treatment regimen did not impact the short-term relapse-free rate of people with ANCA-associated vasculitis (AAV), a post-hoc analysis of ... Read more

March 18, 2020March 21, 2020

Crysvita Normalizes Phosphate Levels, Improves Growth in Children in Real-world Study

XLH News Today

One-year treatment with Crysvita (burosumab) led to the normalization of phosphate levels in children with X-linked hypophosphatemia (XLH) and helped improve their growth, a small real-world study shows. The findings ... Read more

March 17, 2020March 21, 2020

FTO Gene Variants Increase Risk for Extreme Obesity, Brazilian Study Suggests

Genetic Obesity News

Two variants of the FTO gene are strongly associated with the risk of developing extreme obesity, a recent study conducted among the Brazilian population reports. The study, “Genetic variants in ... Read more