Posts by: Patricia Inacio, PhD

News

New nine-month data on four boys with Duchenne muscular dystrophy (DMD) enrolled in Study-101 testing Sarepta Therapeutics‘ micro-dystrophin gene therapy continues to show “very encouraging” results, company officials said. These updated ... Read more

March 29, 2019May 15, 2019

Santhera Filing for EMA Conditional Marketing Authorization for Idebenone to Treat Respiratory Issues in DMD

News

Santhera Pharmaceuticals intends to file for a conditional marketing authorization with the European Medicines Agency (EMA) for idebenone — to be marketed as Puldysa — as a treatment for respiratory ... Read more

March 25, 2019April 18, 2019

Boys with Duchenne Muscular Dystrophy at High Risk for Bone Fractures

News

Young boys with Duchenne muscular dystrophy (DMD) have a higher rate of bone fractures than healthy children, a retrospective study shows. The fractures are more frequent in lower limbs ... Read more

January 30, 2019February 6, 2019

ICER Document Reviews Effectiveness of 3 Duchenne MD Therapies

News

The Institute for Clinical and Economic Review (ICER) has set up a draft document to review evidence of the effectiveness of treatments for Duchenne muscular dystrophy (DMD). The review, “Deflazacort, ... Read more

November 5, 2018November 5, 2018

Pathway Key to Muscle Regeneration Doesn’t Work as It Should in Duchenne, Study Finds

News

The Hippo signaling pathway — a key regulator of tissue development, and cell growth and death — does not work as it should in Duchenne muscular dystrophy and acts to ... Read more

October 12, 2018February 20, 2019

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

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, 2018April 18, 2019

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, 2018February 19, 2019

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

Posts by: Patricia Inacio, PhD

Patricia Inacio, PhD

DMD Gene Therapy Showing ‘Very Encouraging’ Results at 9 Months in Phase 1/2 Study, Sarepta Reports

Santhera Filing for EMA Conditional Marketing Authorization for Idebenone to Treat Respiratory Issues in DMD

Boys with Duchenne Muscular Dystrophy at High Risk for Bone Fractures

ICER Document Reviews Effectiveness of 3 Duchenne MD Therapies

Pathway Key to Muscle Regeneration Doesn’t Work as It Should in Duchenne, Study Finds

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

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