DMD

May 7, 2018May 7, 2018

Capricor Recruiting Duchenne Patients for Phase 2 Trial of CAP-1002

News

Capricor Therapeutics’ Phase 2 clinical trial to evaluate the safety and effectiveness of CAP-1002 in boys and young men with Duchenne muscular dystrophy (DMD) has started recruiting participants. The HOPE-2 ... Read more

April 20, 2018April 20, 2018

#AAN2018 – Clinical Trials Re-Analysis Shows Emflaza Potential to Preserve Motor Function in Boys with DMD

News

Treatment with Emflaza (deflazacort) is better at preserving motor function in patients with Duchenne muscular dystrophy (DMD) than standard of care corticosteroid treatments, according to a re-analysis of data ... Read more

April 8, 2018April 9, 2018

PPMD Names PerkinElmer to Conduct Genetic Tests for Its Decode Duchenne Program

News

Parent Project Muscular Dystrophy (PPMD) has selected PerkinElmer to provide genetic testing for its Decode Duchenne program, which offers testing and counseling at no cost to eligible Duchenne muscular dystrophy (DMD) patients and ... Read more

February 12, 2018

New CRISPR Strategy Corrects Wider Range of Mutations Responsible for DMD

News

A new approach to CRISPR-Cas9 gene-editing technology, called myoediting, successfully restored dystrophin production and contraction force in heart muscle cells of Duchenne muscular dystrophy (DMD) patients. The new strategy, developed ... Read more

January 29, 2018January 29, 2018

European Agency Again Recommends Against Approval of Raxone as DMD Therapy

News

An advisory committee of the European Medicines Agency (EMA) has issued another negative recommendation after re-examining a request to extend Raxone (idebenone) for use in treating Duchenne muscular dystrophy (DMD). The EMA’s ... Read more

January 24, 2018January 24, 2018

DMD Learning Deficits May Be Linked to Cerebellum Impairment, Study Suggests

News

A dysfunctional cerebellum leads to implicit learning deficits in children with Duchenne muscular dystrophy (DMD), Italian researchers have suggested. Their study, “Implicit learning deficit in children with Duchenne muscular ... Read more

January 11, 2018

Analysis of Failed DMD Therapy Kyndrisa Shows Potential Benefit for Younger, Less Impaired Boys

News

Clinical data from a Phase 3 trial revealed that Kyndrisa (drisapersen) could not significantly improve the walking ability of boys with Duchenne muscular dystrophy (DMD) due to mutations amenable ... Read more

January 2, 2018

Exondys 51 Can Preserve Pulmonary Function in Duchenne Patients, Analysis Shows

News

Treatment with Exondys 51 (eteplirsen) can prevent lung function decline in patients with Duchenne muscular dystrophy (DMD), according to the results of an analysis of clinical trial data. The ... Read more

December 21, 2017

New Strategy Developed at UCLA Generates Functional Muscle Cells from Stem Cells in DMD Mice

News

UCLA researchers have developed a new method to efficiently produce and transplant functional skeletal muscle cells from human pluripotent stem cells (hPSCs). This may offer new therapeutic opportunities for ... Read more

October 31, 2017October 31, 2017

Common Flu Virus Could Lead to Muscle Wasting in DMD, Preclinical Study Suggests

News

Infection by the influenza virus, commonly known as the flu, was found to contribute to muscle degeneration in a zebrafish model of Duchenne muscular dystrophy (DMD). Scientists often use zebrafish ... Read more

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