Posts by: Jose Marques Lopes, PhD

News

The European Medicines Agency (EMA) has validated a marketing authorization application for Puldysa (idebenone) for treating respiratory dysfunction in Duchenne muscular dystrophy (DMD) patients who do not take glucocorticoids. This ... Read more

May 10, 2019May 10, 2019

#AANAM — New TTN Gene Variants Potentially Linked to Limb-girdle MD, Study Says

News

Limb-girdle muscular dystrophy (LGMD) may be caused by previously unidentified TTN gene variants that are also associated with heart muscle disease, according to a study. Findings of the study, ... Read more

May 3, 2019

EGF Protein Preserves Muscle Strength, Promotes Muscle Regeneration in DMD Mice, Study Shows

News

A protein called epidermal growth factor (EGF) can help preserve muscle strength and increase muscle regeneration in a mouse model of Duchenne muscular dystrophy (DMD), a finding that may ... Read more

April 15, 2019June 28, 2019

Audentes Therapeutics Expanding Treatment Candidates for Duchenne MD and Myotonic Dystrophy Type 1

News

Audentes Therapeutics is expanding its pipeline of potential molecular therapies, expecting to address 80% of patients with Duchenne muscular dystrophy (DMD) and all with myotonic dystrophy type 1 (DM1). ... Read more

April 10, 2019June 21, 2019

Raising Both Dystrophin and Utrophin Levels May Rescue Muscles in DMD, Mouse Study Suggests

News

Using a genetic engineering tool to restore dystrophin while raising levels of utrophin, a similar protein, leads to better improvements in muscle function than either approach alone, research in ... Read more

March 8, 2019March 22, 2019

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

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

Posts by: Jose Marques Lopes, PhD

Jose Marques Lopes, PhD

European Agency OKs Marketing Authorization for Puldysa to Treat Respiratory Decline in Duchenne MD

#AANAM — New TTN Gene Variants Potentially Linked to Limb-girdle MD, Study Says

EGF Protein Preserves Muscle Strength, Promotes Muscle Regeneration in DMD Mice, Study Shows

Audentes Therapeutics Expanding Treatment Candidates for Duchenne MD and Myotonic Dystrophy Type 1

Raising Both Dystrophin and Utrophin Levels May Rescue Muscles in DMD, Mouse Study Suggests

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

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