Posts by: Patricia Inacio, PhD

News

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to investigational therapy Sarconeos for Duchenne muscular dystrophy (DMD), Biophytis, the therapy’s developer, announced. The company also has filed ... Read more

May 14, 2018May 14, 2018

New Exon 51 Skipping Therapy in Phase 1 Safety Trial Explained by Sarepta CEO in Webinar

News

Exondys 51 (eteplirsen), an RNA-targeted therapy marketed by Sarepta Therapeutics, is the first approved treatment targeting the underlying cause of Duchenne muscular dystrophy (DMD). Sarepta is continuing to refine its RNA-targeted ... Read more

April 30, 2018April 30, 2018

#AAN2018 – 1 Year of Edasalonexent Use Significantly Slows DMD Progression, MoveDMD Trial Reports

News

The investigational therapy edasalonexent was seen to slow disease progression in boys with Duchenne muscular dystrophy, almost one-year results from the MoveDMD trial report. Specifically, edasalonexent — administered orally ... Read more

March 28, 2018March 28, 2018

Tiny Spear-like Nanotubes May Enhance Delivery of Gene Therapies, Study Suggests

News

Magnetic nanotubes with a spear-like tip may enhance the precision and effectiveness of gene therapy delivery, a promising therapeutic strategy for many genetic diseases, including muscular dystrophy, according to UCLA researchers. ... Read more

March 19, 2018March 19, 2018

Expert Uses Webinar to Explain the Science Behind Gene Therapies for Duchenne MD

News

Scientists are increasingly looking at gene therapies as potential treatments for Duchenne muscular dystrophy. But there are a lot of ways to do them, and an expert in the field ... Read more

March 9, 2018

Summit Therapeutics Expands PhaseOut DMD Clinical Trial of Ezutromid

News

Summit Therapeutics has opened enrollment for an additional group in its ongoing PhaseOut DMD trial to include patients with Duchenne muscular dystrophy (DMD) who participated in the previous Phase 1 clinical ... Read more

March 1, 2018

Ezutromid Significantly Reduces Muscle Inflammation, Interim Analysis of PhaseOut DMD Trial Shows

News

A new interim analysis of the PhaseOut DMD clinical trial shows that ezutromid (SMT C1100) significantly decreased muscle inflammation in patients with Duchenne muscular dystrophy (DMD), Summit Therapeutics announced. Ezutromid ... Read more

February 21, 2018February 21, 2018

Phase 3 Trial of Translarna’s Long-term Efficacy Enrolling Duchenne MD Patients with Nonsense Mutations

News

PTC Therapeutics is recruiting Duchenne muscular dystrophy (DMD) patients ages 5 and older and with a nonsense mutation for a long-term Phase 3 study to characterize the effects of ... Read more

February 19, 2018

New Device Measures Cell Strength Faster, More Accurately, Aiding in Search for MD Therapies

News

Measuring the physical strength of individual cells is now 100 times faster with a new device called FLECS developed by researchers at UCLA and Rutgers University. FLECS allows researchers ... Read more

February 5, 2018February 5, 2018

Researchers Use Artificial Chromosomes to Deliver Gene Therapy for DMD

News

European and Japanese researchers used an artificial chromosome to generate the dystrophin protein that’s missing in Duchenne muscular dystrophy. The chromosome delivered a two-gene package capable of producing the ... Read more

Posts by: Patricia Inacio, PhD

Patricia Inacio, PhD

Myoda (Sarconeos) Earns FDA’s Orphan Drug Designation for Duchenne MD

New Exon 51 Skipping Therapy in Phase 1 Safety Trial Explained by Sarepta CEO in Webinar

#AAN2018 – 1 Year of Edasalonexent Use Significantly Slows DMD Progression, MoveDMD Trial Reports

Tiny Spear-like Nanotubes May Enhance Delivery of Gene Therapies, Study Suggests

Expert Uses Webinar to Explain the Science Behind Gene Therapies for Duchenne MD

Summit Therapeutics Expands PhaseOut DMD Clinical Trial of Ezutromid

Ezutromid Significantly Reduces Muscle Inflammation, Interim Analysis of PhaseOut DMD Trial Shows

Phase 3 Trial of Translarna’s Long-term Efficacy Enrolling Duchenne MD Patients with Nonsense Mutations

New Device Measures Cell Strength Faster, More Accurately, Aiding in Search for MD Therapies

Researchers Use Artificial Chromosomes to Deliver Gene Therapy for DMD

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