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.

June 25, 2020June 25, 2020

Phase 1 Trial of Potential Oral Treatment for LGMD2i Doses 1st Volunteer

News

A Phase 1 clinical trial of BBP-418, a potential first oral therapy for limb-girdle muscular dystrophy type 2i (LGMD2i), has dosed its first healthy volunteer, ML Bio Solutions announced. The company also ... Read more

May 28, 2020May 30, 2020

Viltolarsen Raises Dystrophin Levels, Aids Muscles in DMD Boys in Phase 2 Trial

News

NS Pharma’s lead candidate viltolarsen safely and effectively increases dystrophin levels and promotes improvements in boys with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping, final data from a Phase 2 trial show. “As ... Read more

May 19, 2020May 19, 2020

Pfizer’s DMD Gene Therapy Continues to Show Promise, Phase 1b Trial Shows

News

Pfizer’s experimental gene therapy PF-06939926 promotes sustained production of a shorter but functional version of dystrophin — the protein lacking in Duchenne muscular dystrophy (DMD) — and improves motor ... Read more

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

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