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.

March 20, 2020March 20, 2020

Viltolarsen Available to U.S. DMD Patients Under Expanded Access Program

News

NS Pharma launched an expanded access program (EAP) in the U.S. to allow certain Duchenne muscular dystrophy (DMD) patients to gain access to its investigational exon-skipping therapy viltolarsen. The program will allow physicians to prescribe ... Read more

March 13, 2020March 13, 2020

MDA Let’s Play Uses Gaming to Raise Funds for Neuromuscular Disorders

News

The Muscular Dystrophy Association (MDA) is launching MDA Let’s Play, a platform to raise funding and awareness about muscular dystrophy and related neuromuscular disorders through gaming. “We’ve always been an iconic ... Read more

January 30, 2020January 30, 2020

Cx43 Protein May Be Key to Preventing Heart Disease in DMD, Mouse Study Suggests

News

Altering connexin 43 (Cx43) — a protein involved in heart function — in cardiac muscle cells protected against heart disease in a mouse model of Duchenne muscular dystrophy (DMD), ... Read more

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, 2019March 27, 2020

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

January 28, 2019January 28, 2020

Sarepta Joins Roche to Speed DMD Gene Therapy Development, Access Outside US

News

Sarepta Therapeutics is partnering with Roche to accelerate the development and expand the availability of SRP-9001 — its leading gene therapy candidate for Duchenne muscular dystrophy (DMD) — outside the ... Read more

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