Posts by: Patricia Inacio, PhD

Patricia Inacio, PhD

Patricia holds a Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She has also served as a PhD student research assistant at the Department of Microbiology & Immunology, Columbia University, New York.

October 9, 2019October 9, 2019

Gene Therapy SRP-9003 Showing ‘Very Encouraging’ Results at 9 Months in Limb Girdle MD, Sarepta Reports

News

New nine-month data from a Phase 1/2 trial show that a single administration of the gene therapy SRP-9003 (formerly, MYO-101) at low dose significantly improved functional measures and lowered the levels of ... Read more

September 18, 2019October 2, 2019

Acceleron Stops Clinical Development of ACE-083 as Treatment for FSHD

News

Due to a lack of efficacy, Acceleron Pharma is halting the clinical development of its ACE-083 candidate for the treatment of facioscapulohumeral muscular dystrophy (FSHD) . Topline results from a ... Read more

September 13, 2019October 2, 2019

Microdystrophin Gene Therapy Improves with nNOS Domain, Review Study Says

News

A review study highlights the benefits of adding an extra protein domain — called neuronal nitric oxide synthase (nNOS) — to microdystrophin gene therapy. nNOS is an important player ... Read more

September 11, 2019September 25, 2019

MDA Awards $6.6M to 25 Scientists Working in DMD, Other Neuromuscular Diseases

News

Twenty-five research grants totaling more than $6.6 million have been awarded by the Muscular Dystrophy Association (MDA) to support studies into the mechanisms of neuromuscular diseases, including Duchenne and ... Read more

August 23, 2019September 11, 2019

FDA Raises Concerns About Golodirsen as Potential Exon 53 Skipping Duchenne Therapy

News

The U.S Food and Drug Administration (FDA) has rejected Sarepta Therapeutic’s application seeking accelerated approval of golodirsen (SRP-4053) for Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping. Golodirsen (SRP-4053) ... Read more

July 15, 2019July 15, 2019

Exondys 51 Superior to Glucocorticoids in Preserving Respiratory Health of Duchenne Boys, Analysis Shows

News

Treatment with Exondys 51 (eteplirsen) significantly slows respiratory decline in boys with Duchenne muscular dystrophy (DMD), including those with more advanced disease, compared to standard glucocorticoid therapy, an analysis of clinical trials ... Read more

May 31, 2019August 2, 2019

Santhera Seeks European Approval of Puldysa to Address Respiratory Problems in DMD

News

Santhera Pharmaceuticals has submitted a marketing authorization request to the European Medicines Agency (EMA) for Puldysa (idebenone) as a treatment for respiratory dysfunction in patients with Duchenne muscular dystrophy (DMD). The ... Read more

April 3, 2019June 3, 2019

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

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

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