Posts by: Jose Marques Lopes, PhD

News

All three children with limb girdle muscular dystrophy type 2E (LGMD2E) treated with the investigational gene therapy MYO-101 in a clinical trial showed marked production of beta-sarcoglycan, the missing protein ... Read more

March 1, 2019March 12, 2019

Edasalonexent May Limit Disease Progression in Boys with Duchenne MD, Phase 1/2 Trial Shows

News

The investigational treatment edasalonexent was well-tolerated, led to no safety issues, and blocked a crucial muscle disease protein in boys with Duchenne muscular dystrophy (DMD), according to part 1 ... Read more

February 25, 2019March 8, 2019

New Device Enables Home Monitoring of Respiratory Function in Teens with DMD, Study Reports

News

A new hand-held device makes monitoring pulmonary function in adolescents with Duchenne muscular dystrophy (DMD) more convenient and may improve disease management, a study shows. The study, “Home-Based Monitoring ... Read more

February 22, 2019March 8, 2019

Edasalonexent Leads to Normal Growth, Other Benefits in Boys with DMD, Phase 1/2 Trial Shows

News

Treatment with investigational edasalonexent enabled normal growth, preserved muscle function, and slowed disease progression in boys with Duchenne muscular dystrophy (DMD), according to results of a Phase 1/2 trial and ... Read more

January 28, 2019February 12, 2019

Phase 2b Trial Testing Vamorolone Now Enrolling Boys Ages 4-6 with DMD

News

A Phase 2b trial of the investigational therapy vamorolone is 4-to-6-year-old boys with Duchenne muscular dystrophy (DMD) has begun to enroll patients. Patient recruitment for the international ReveraGen BioPharma-sponsored, double-blind ... Read more

January 27, 2019March 12, 2019

Cell Therapy CAP-1002 Leads to Heart, Skeletal Muscle Improvements in DMD Patients, Phase 1/2 Trial Shows

News

Treatment with investigational cell therapy CAP-1002 improved heart and skeletal muscle function, and reduced cardiac scarring in boys and young men with advanced Duchenne muscular dystrophy (DMD) in a ... Read more

January 9, 2019January 9, 2019

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

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

Posts by: Jose Marques Lopes, PhD

Jose Marques Lopes, PhD

MYO-101 Enables Production of Missing Protein in Children with LGMD2E, Trial Shows

Edasalonexent May Limit Disease Progression in Boys with Duchenne MD, Phase 1/2 Trial Shows

New Device Enables Home Monitoring of Respiratory Function in Teens with DMD, Study Reports

Edasalonexent Leads to Normal Growth, Other Benefits in Boys with DMD, Phase 1/2 Trial Shows

Phase 2b Trial Testing Vamorolone Now Enrolling Boys Ages 4-6 with DMD

Cell Therapy CAP-1002 Leads to Heart, Skeletal Muscle Improvements in DMD Patients, Phase 1/2 Trial Shows

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

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