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.

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

November 8, 2019November 8, 2019

Experimental Cell Therapy Improves Cardiac Function in Mouse Model of DMD, Study Says

News

Treatment with a cell therapy candidate called dystrophin expressing chimeras (DECs) increased dystrophin levels in heart muscle and improved cardiac function in mice with Duchenne muscular dystrophy (DMD), a ... Read more

October 9, 2019October 9, 2019

Gene Therapy SRP-9003 Showing ‘Very Encouraging’ Results at 9 Months in Limb Girdle MD, Sarepta Reports

News

New nine-month data from a Phase 1/2 trial show that a single administration of the gene therapy SRP-9003 (formerly, MYO-101) at low dose significantly improved functional measures and lowered the levels of ... Read more

September 18, 2019October 2, 2019

Acceleron Stops Clinical Development of ACE-083 as Treatment for FSHD

News

Due to a lack of efficacy, Acceleron Pharma is halting the clinical development of its ACE-083 candidate for the treatment of facioscapulohumeral muscular dystrophy (FSHD) . Topline results from a ... Read more

September 13, 2019October 2, 2019

Microdystrophin Gene Therapy Improves with nNOS Domain, Review Study Says

News

A review study highlights the benefits of adding an extra protein domain — called neuronal nitric oxide synthase (nNOS) — to microdystrophin gene therapy. nNOS is an important player ... Read more

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