Posts by: Jose Marques Lopes, PhD

News

A Phase 3 trial of edasalonexent, an oral disease-modifying treatment candidate, is enrolling boys with Duchenne muscular dystrophy (DMD) at multiple sites in the U.S. and will soon open ... Read more

January 2, 2019January 2, 2019

CureDuchenne and Avidity Biosciences Hosting Webinar Friday on Potential DMD Therapies

News

CureDuchenne and Avidity Biosciences will host a webinar Friday to brief the Duchenne muscular dystrophy (DMD) community on Avidity’s preclinical development of DMD therapy candidates. The webinar will take ... Read more

September 19, 2018September 19, 2018

Potential DMD Therapy Vamorolone Shows Positive Effects in Phase 2a Trial, Study Reports

News

Vamorolone, a Duchenne muscular dystrophy (DMD) treatment candidate, showed anti-inflammatory activity and an improved safety profile over the glucocorticoid prednisolone in a Phase 2a clinical trial for boys 4 ... Read more

September 14, 2018September 14, 2018

Gene Editing Restored Dystrophin Levels in Dog Model of Duchenne MD, Study Reports

News

Using the gene editing process CRISPR/Cas9 enabled a marked restoration of dystrophin production in various muscles of dogs with Duchenne muscular dystrophy (DMD). The study with that finding, “Gene editing ... Read more

July 23, 2018July 23, 2018

Raxone Delays Assisted Ventilation in DMD Patients by 3 Years, Phase 3 Trial Results Show

News

Treatment of Duchenne muscular dystrophy (DMD) patients with Raxone (idebenone) delays the need for assisted ventilation by three years, new data from a Phase 3 clinical trial show. Trial ... Read more

July 9, 2018July 10, 2018

Potential DMD Therapy Now in Phase 1 Trial, WVE-210201, Likely To Be Named Orphan Drug in EU

News

The Committee for Orphan Medicinal Products (COMP), an arm of the European Medicines Agency (EMA), is recommending that WVE-210201 be designated an orphan drug as a potential Duchenne muscular dystrophy (DMD) ... Read more

June 27, 2018June 27, 2018

UK Agency Renews Early Access Program for Raxone to Treat Duchenne MD

News

People with Duchenne muscular dystrophy (DMD) in the U.K. will continue to have access to Raxone (idebenone) to treat their disease, after the Medicines and Healthcare products Regulatory Agency (MHRA) renewed its ... Read more

June 25, 2018June 25, 2018

Way to Accurately Measure Changes in Ankle Muscles in Myotonic Dystrophy Type 1 May Aid Clinical Trials

News

A quantitative method that can detect changes in ankle muscle strength in patients with myotonic dystrophy type 1 (MD1) was found to be highly accurate, and may work as an ... Read more

June 15, 2018June 15, 2018

Proteins That Regulate Gene with Critical Role in FSHD Development Identified in Study

News

Researchers identified proteins that regulate DUX4, the key gene in facioscapulohumeral muscular dystrophy (FSHD). The results offer a potential therapeutic pathway, they believe. The study, “NuRD and CAF-1-mediated silencing ... Read more

Posts by: Jose Marques Lopes, PhD

Jose Marques Lopes, PhD

Phase 3 Trial of Edasalonexent in DMD Now Enrolling Across US, Global Sites to Follow

Top 10 Muscular Dystrophy Articles of 2018

CureDuchenne and Avidity Biosciences Hosting Webinar Friday on Potential DMD Therapies

Potential DMD Therapy Vamorolone Shows Positive Effects in Phase 2a Trial, Study Reports

Gene Editing Restored Dystrophin Levels in Dog Model of Duchenne MD, Study Reports

Raxone Delays Assisted Ventilation in DMD Patients by 3 Years, Phase 3 Trial Results Show

Potential DMD Therapy Now in Phase 1 Trial, WVE-210201, Likely To Be Named Orphan Drug in EU

UK Agency Renews Early Access Program for Raxone to Treat Duchenne MD

Way to Accurately Measure Changes in Ankle Muscles in Myotonic Dystrophy Type 1 May Aid Clinical Trials

Proteins That Regulate Gene with Critical Role in FSHD Development Identified in Study

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