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.

November 26, 2020November 26, 2020

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

News, syndicated

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to ... Read more

November 3, 2020November 3, 2020

PPMD Seeking Adults with Duchenne or Becker MD to Join Advisory Committee

News

Parent Project Muscular Dystrophy (PPMD) is seeking adults with Duchenne or Becker muscular dystrophy to serve on the 2021 PPMD Adult Advisory Committee (PAAC). The committee is meant to ... Read more

October 29, 2020October 29, 2020

Phase 3 Trial Failure Prompts Catabasis to Stop Edasalonexent Development

News

Catabasis Pharmaceuticals’ investigational oral therapy edasalonexent failed to preserve muscle function and overall functionality in boys with Duchenne muscular dystrophy (DMD), top-line data from the PolarisDMD Phase 3 clinical trial ... Read more

October 8, 2020October 8, 2020

Pfizer’s Gene Therapy for Duchenne Placed on Fast Track by FDA

News

The U.S. Food and Drug Administration (FDA) has granted fast track designation to PF-06939926, Pfizer’s investigational gene therapy for Duchenne muscular dystrophy (DMD). This designation is given to treatments ... Read more

October 6, 2020October 6, 2020

FDA Lifts Clinical Hold on IGNITE DMD Gene Therapy Trial

News

The U.S. Food and Drug Administration (FDA) has lifted its clinical hold on the Phase 1/2 trial testing SGT-001, Solid Biosciences’ investigational gene therapy for people with Duchenne muscular ... Read more

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, 2020October 8, 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