Posts by: Patricia Inacio, PhD

News

A global Phase 3 trial evaluating the efficacy and safety of oral edasalonexent in patients with Duchenne muscular dystrophy (DMD) will begin enrolling boys, ages 4 to 7, in the coming ... Read more

June 29, 2018July 2, 2018

Summit Therapeutics Ends Development of Ezutromid Therapy for DMD After Trial Failure

News

Summit Therapeutics is stopping the clinical development of ezutromid, which it was investigating as a Duchenne muscular dystrophy (DMD) treatment, after a Phase 2 clinical trial testing the therapy failed to ... Read more

June 20, 2018June 20, 2018

Clinical Hold on Duchenne Gene Therapy Trial Lifts, IGNITE DMD Again Enrolling Patients

News

The U.S. Food and Drug Administration (FDA) has lifted its clinical hold on the Phase 1/2 trial testing SGT-001, a dystrophin gene therapy aiming to treat Duchenne muscular dystrophy (DMD). FDA, in ... Read more

June 18, 2018June 18, 2018

Rehab Is Still Major Goal in New, Updated CDC Guidelines for Duchenne Management

News

The Centers for Disease Control and Prevention (CDC) released new, updated guidelines for the management of Duchenne muscular dystrophy (DMD) patients. Researchers highlighted the role of rehabilitation techniques and ... Read more

June 11, 2018June 11, 2018

FDA Names Potential FSHD Therapy GBC0905 an Orphan Drug to Speed Its Development

News

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to GBC0905 as a potential treatment for facioscapulohumeral muscular dystrophy (FSHD),its developer, Genea Biocells, announced. Orphan drug status aims to ... Read more

June 8, 2018June 8, 2018

CHMP Opposes Approval of Exondys 51 to Treat DMD in Europe

News

The Committee for Medicinal Products for Human Use (CHMP) — part of the European Medicines Agency (EMA) — is recommending against Exondys 51 (eteplirsen) as a therapy for Duchenne muscular dystrophy (DMD) patients ... Read more

May 18, 2018May 18, 2018

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

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

Posts by: Patricia Inacio, PhD

Patricia Inacio, PhD

Phase 3 Trial of Oral Edasalonexent as Potential DMD Treatment to Open Soon, Catabasis Announces

Summit Therapeutics Ends Development of Ezutromid Therapy for DMD After Trial Failure

Clinical Hold on Duchenne Gene Therapy Trial Lifts, IGNITE DMD Again Enrolling Patients

Rehab Is Still Major Goal in New, Updated CDC Guidelines for Duchenne Management

FDA Names Potential FSHD Therapy GBC0905 an Orphan Drug to Speed Its Development

CHMP Opposes Approval of Exondys 51 to Treat DMD in Europe

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

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