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.

February 25, 2021February 25, 2021

Exon Skipping Dominates DMD Treatment, But What’s on Horizon?

News

With the recent approval of Amondys 45 (casimersen) by Sarepta Therapeutics, there are now four exon-skipping therapies available in the U.S. for eligible patients with Duchenne muscular dystrophy (DMD). The others are Exondys ... Read more

February 25, 2021February 26, 2021

FDA Approves Amondys 45 for Duchenne Patients With Exon 45 Mutation

News

The U.S. Food and Drug Administration (FDA) has conditionally approved Amondys 45 — formerly known as casimersen — by Sarepta Therapeutics as the first treatment for people with Duchenne muscular dystrophy (DMD) amenable to exon 45 ... Read more

February 19, 2021February 19, 2021

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

News, syndicated

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare ... Read more

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 a ... 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 represent ... 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 show. ... 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 that ... 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 dystrophy ... 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 announced ... Read more

May 28, 2020February 19, 2021

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 a ... Read more