Posts by: Jose Marques Lopes, PhD

News

A genetic compensation mechanism discovered in zebra fish could have implications in the treatment of muscle diseases, a recent study suggests. The study, “Genetic compensation triggered by actin mutation ... Read more

April 2, 2018April 2, 2018

Tamoxifen and Evista Improved Muscle, Other MD Functions in Mouse Study

News

The use of tamoxifen and Evista (raloxifene) improved cardiac, respiratory, and skeletal muscle functions, and increased bone density in a mouse model of muscular dystrophy (MD), research from the Carolinas ... Read more

March 21, 2018March 21, 2018

AMO Pharma’s Myotonic Dystrophy Therapy Improves Cognition and Function, Phase 2 Trial Shows

News

AMO Pharma’s myotonic dystrophy treatment improved patients’ cognition, fatigue and ability to perform daily tasks, a Phase 2 clinical trial showed. The therapy, AMO-02 (tideglusib), also improved patients’ autism symptoms. In addition, it ... Read more

March 14, 2018

Sarepta to Seek Accelerated US Approval of Duchenne MD Therapy Golodirsen

News

Sarepta Therapeutics will seek accelerated U.S. approval of a treatment for a subset of Duchenne muscular dystrophy (DMD) patients. Golodirsen (SRP-4053) is for patients whose disease stems from a deleted part of ... Read more

March 12, 2018March 12, 2018

Vietnamese Wins Kovalevskaya Award for Prenatal Diagnosis of Duchenne MD

News

Research supporting prenatal diagnosis of Duchenne muscular dystrophy (DMD) has earned Vietnamese scientist Tran Van Khanh a Kovalevskaya Award. Khanh has focused her work on studying gene therapy and ... Read more

February 23, 2018February 23, 2018

Duchenne MD Patients with Respiratory Decline Gain Access to Raxone in US

News

The investigational treatment Raxone (idebenone) could become available to qualifying Duchenne muscular dystrophy (DMD) patients in the U.S. with respiratory decline following the start of an expanded access program ... Read more

February 14, 2018

Catabasis Announces Global Phase 3 Trial of Edasalonexent for DMD Boys

News

Treatment with edasalonexent (CAT-1004) showed sustained preservation of muscle function in a Phase 1/2 trial in boys with Duchenne muscular dystrophy (DMD), according to Catabasis Pharmaceuticals. The MoveDMD trial (NCT02439216) ... Read more

February 9, 2018

Bodysuit Will Use Artificial Intelligence to Track Movement in DMD Patients

News

The U.K.-based Duchenne Research Fund has granted £320,000 ($441,760) to researchers at Imperial College London to develop a bodysuit that may improve treatment decisions in Duchenne muscular dystrophy (DMD). The ... Read more

February 2, 2018February 2, 2018

Skyhawk Secures $8M in Seed Funding to Advance Small Molecule Therapies Targeting RNA

News

The recently formed company Skyhawk Therapeutics announced it has obtained $8 million in seed funding to develop RNA-targeting small molecules for the treatment of over 50 diseases, including muscular dystrophy. ... Read more

January 9, 2018

Acceleron Therapy Increases Facioscapulohumeral Dystrophy Patients’ Muscle Mass, Trial Shows

News

Acceleron Pharma’s treatment for a muscular dystrophy affecting the face and other muscles increased the muscle mass of those with the disorder, according to a Phase 2 clinical trial. The ... Read more

Posts by: Jose Marques Lopes, PhD

Jose Marques Lopes, PhD

Genetic Compensation Mechanism Could Aid in Treatment of Muscle Disorders, Study Suggests

Tamoxifen and Evista Improved Muscle, Other MD Functions in Mouse Study

AMO Pharma’s Myotonic Dystrophy Therapy Improves Cognition and Function, Phase 2 Trial Shows

Sarepta to Seek Accelerated US Approval of Duchenne MD Therapy Golodirsen

Vietnamese Wins Kovalevskaya Award for Prenatal Diagnosis of Duchenne MD

Duchenne MD Patients with Respiratory Decline Gain Access to Raxone in US

Catabasis Announces Global Phase 3 Trial of Edasalonexent for DMD Boys

Bodysuit Will Use Artificial Intelligence to Track Movement in DMD Patients

Skyhawk Secures $8M in Seed Funding to Advance Small Molecule Therapies Targeting RNA

Acceleron Therapy Increases Facioscapulohumeral Dystrophy Patients’ Muscle Mass, Trial Shows

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