Posts by: Patricia Inacio, PhD

Patricia Inacio, PhD

Patricia holds a Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She has also served as a PhD student research assistant at the Department of Microbiology & Immunology, Columbia University, New York.

September 8, 2020September 8, 2020

Santhera Acquires Global Rights to DMD Therapy Vamorolone

News

Santhera Pharmaceuticals has obtained the worldwide rights to vamorolone (VBP15), an experimental therapy for Duchenne muscular dystrophy (DMD), the company announced. The company has reached an agreement with ReveraGen Biopharma, ... Read more

September 1, 2020September 1, 2020

Virtual Events Mark MDA’s Muscular Dystrophy Awareness Month

News

This year, the Muscular Dystrophy Association (MDA) will celebrate MDA National Muscular Dystrophy Awareness Month, held every September, with a series of online events. The events will be held virtually ... Read more

August 4, 2020August 4, 2020

FDA Requests More Data, Maintains Clinical Hold on IGNITE DMD Trial of SGT-001 Gene Therapy

News

The U.S. Food and Drug Administration (FDA) is maintaining the clinical hold on the Phase 1/2 trial testing Solid Biosciences’ gene therapy SGT-001 for people with Duchenne muscular dystrophy (DMD), the company ... Read more

May 14, 2020May 14, 2020

CAP-1002 Improves Muscle, Lung and Heart Function in Young DMD Patients, HOPE-2 Trial Data Show

News

One-year treatment with CAP-1002, Capricor Therapeutics’ investigational cell therapy for Duchenne muscular dystrophy (DMD) significantly improves respiratory, cardiac and upper limb function in boys and young men at advanced stages of the ... Read more

March 16, 2020March 16, 2020

Vyondys 53 Boosts Dystrophin Production in Boys With DMD, Early Results Show

News

Treatment with Vyondys 53 (golodirsen) for 48 weeks increases levels of dystrophin protein by 16-fold in boys with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping, updated results of a Phase ... Read more

March 12, 2020March 12, 2020

Increlex Improves Growth, But Not Muscle Function in DMD, Early Trial Shows

News

Treatment for six months with Increlex (mecasermin), an injectable form of insulin-like growth factor-I (IGF-I), enhanced growth in boys with Duchenne muscular dystrophy (DMD), a Phase 1/2 trial shows. ... Read more

March 10, 2020March 10, 2020

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

Muscular Dystrophy News, News

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still ... Read more

March 3, 2020March 3, 2020

Small Molecule Raises Utrophin Levels to Rescue Muscle Function in DMD, Mouse Study Suggests

News

A small molecule, trichostatin A (TSA), increased the levels of a dystrophin-related protein known as utrophin, and improved the structure and function of muscles in a mouse model of Duchenne ... Read more

December 11, 2019December 11, 2019

GPM-owned Convenience Stores Run Holiday Campaign to Support Muscular Dystrophy Association

News

GPM Investments, the sixth largest convenience store chain in the U.S., is running its “Tis the Season for Giving” holiday campaign throughout December to support the Muscular Dystrophy Association ... Read more

December 6, 2019January 15, 2020

Mitochondrial Protein’s Loss Linked to Muscle Weakness and Poor Repair in Early Study

News

Loss of MICU1, a key regulator of calcium balance in cells, was seen to affect muscle function and repair in a study in mice and cells from people lacking ... Read more