Posts by: Jose Marques Lopes, PhD

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

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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, 2019April 15, 2019

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

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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, 2019April 10, 2019

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

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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

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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

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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

Posts by: Jose Marques Lopes, PhD

Jose Marques Lopes, PhD

#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

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

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