Posts by: Patricia Inacio, PhD

News

Sarepta Therapeutics‘ microdystrophin gene therapy enhanced dystrophin protein expression in the muscles of four boys with Duchenne muscular dystrophy (DMD) enrolled in a Phase 1/2 trial. More importantly, the ... Read more

September 26, 2018September 26, 2018

FDA Lifts Hold on Sarepta’s Phase 1/2 Trial on Gene Therapy for DMD

News

The U.S. Food and Drug Administration has lifted the clinical hold on a Phase 1/2 trial evaluating Sarepta Therapeutics‘ micro-dystrophin gene therapy candidate for Duchenne muscular dystrophy (DMD), the company announced in a press ... Read more

September 21, 2018September 21, 2018

Nonprofit Coalition to Cure Calpain 3 Funds 2 Gene Therapy Research Projects for LGMD2A

News

Two new research projects have been funded by the Coalition to Cure Calpain 3 (C3), a nonprofit patient advocacy organization, to develop gene therapies for limb-girdle muscular dystrophy type ... Read more

September 12, 2018September 12, 2018

STRIVE Awards Recognize Efforts of Duchenne MD Patient Groups Worldwide

News

PTC Therapeutics announced the winners of the 2018 global Strategies to Realize Innovation, Vision and Empowerment (STRIVE) awards, which recognize the efforts of nonprofit organizations working to serve the ... Read more

July 13, 2018July 13, 2018

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

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

Posts by: Patricia Inacio, PhD

Patricia Inacio, PhD

Sarepta’s Gene Therapy Improves Muscle Function in 4 Boys with DMD, Phase 1/2 Trial Shows

FDA Lifts Hold on Sarepta’s Phase 1/2 Trial on Gene Therapy for DMD

Nonprofit Coalition to Cure Calpain 3 Funds 2 Gene Therapy Research Projects for LGMD2A

STRIVE Awards Recognize Efforts of Duchenne MD Patient Groups Worldwide

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

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