Posts by: Marta Figueiredo

Marta Figueiredo

Marta Figueiredo holds a BSc in Biology and a MSc in Evolutionary and Developmental Biology from the University of Lisbon, Portugal. She is currently finishing her PhD in Biomedical Sciences at the University of Lisbon, where she focused her research on the role of several signalling pathways in thymus and parathyroid glands embryonic development.

December 20, 2019January 8, 2020

Vyondys 53 Available to Duchenne Patients in the U.S.

News

The newly approved therapy Vyondys 53 (golodirsen) is available for immediate distribution to treat people with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping, according to the medicine’s developer Sarepta Therapeutics. ... Read more

December 13, 2019December 13, 2019

FDA, in Reversal, Approves Vyondys 53 to Treat Duchenne Patients with Exon 53 Mutations

News

The U.S. Food and Drug Administration has conditionally approved Vyondys 53 (golodirsen) to treat Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping. This accelerated approval comes less than ... Read more

November 4, 2019November 4, 2019

BIIB100 Improves Motor Function, Slows DMD Progression in Animal Models, Study Finds

News

An experimental therapy called BIIB100 improved motor function and slowed the progression of Duchenne muscular dystrophy (DMD) in animal models of the disease, a study shows. These findings suggest ... Read more

October 28, 2019October 28, 2019

Vamorolone Designated Promising Innovative Medicine in UK for DMD Treatment

News

The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted promising innovative medicine (PIM) designation to ReveraGen’s vamorolone for the treatment of Duchenne muscular dystrophy (DMD). PIM designation indicates that ... Read more

October 4, 2019November 13, 2019

First Baby Tested for Duchenne in PPMD’s Newborn Screening Pilot Program in NY

News

Parent Project Muscular Dystrophy (PPMD) announced the screening of a first baby for Duchenne muscular dystrophy (DMD) within the organization’s pilot newborn screening program in New York. The $5 million pilot ... Read more

September 16, 2019October 2, 2019

New Blood Biomarkers of DMD and Glucocorticoid Response Identified

News

Researchers have identified a new set of blood biomarkers of Duchenne muscular dystrophy (DMD) and of response to standard glucocorticoid therapy. The study, conducted by U.S. and Canadian researchers, ... Read more

November 2, 2018November 2, 2018

Capricor’s Vesicle-based Therapy Holds Promise to Treat DMD, Preclinical Data Suggests

News

Capricor Therapeutics’ vesicle-based therapy CAP-2003, derived from the company’s proprietary cardiosphere-derived cells (CDCs), may be an effective therapy for Duchenne muscular dystrophy (DMD), according to preclinical data. The company ... Read more

June 22, 2018February 25, 2019

Microdystrophin Gene Therapy Shows Promising Interim Results in Phase 1/2 Trial

News

Microdystrophin gene therapy robustly induces the production of a shorter, but functional, version of the dystrophin protein and reduces muscle damage in Duchenne muscular dystrophy (DMD) patients, according to ... Read more

February 12, 2018April 18, 2019

New CRISPR Strategy Corrects Wider Range of Mutations Responsible for DMD

News

A new approach to CRISPR-Cas9 gene-editing technology, called myoediting, successfully restored dystrophin production and contraction force in heart muscle cells of Duchenne muscular dystrophy (DMD) patients. The new strategy, developed ... Read more

January 26, 2018

Ezutromid Reduces Muscle Damage in DMD Patients, Interim Data of Phase 2 Trial Shows

News

Ezutromid significantly reduced muscle damage in patients with Duchenne muscular dystrophy (DMD) after 24 weeks of treatment in a Phase 2 trial, Summit Therapeutics recently announced. DMD is a severe ... Read more

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