Posts by: Joana Carvalho

News

Santhera Pharmaceuticals has entered into a collaboration agreement with researchers from the Biozentrum of the University of Basel in Switzerland to develop a new gene therapy for treating people ... Read more

May 22, 2019May 22, 2019

PPMD Awards $100K for Immune Cell Profiling Research for Duchenne and Becker at UCLA

News

The Parent Project Muscular Dystrophy (PPMD) has awarded a $100,000 grant to a UCLA professor to support research on the signature profile of immune cells in patients with Duchenne and ... Read more

May 1, 2019May 1, 2019

Lab Test Allows Prenatal Diagnosis of One Type of Congenital MD, Study Finds

News

Merosin-deficient muscular dystrophy, a type of congenital MD, might be diagnosed before birth using immunohistochemistry, an easy and quick lab test that looks for specific markers in cells from a ... Read more

April 24, 2019June 28, 2019

Wave’s Exon 51-Skipping Therapy Safe, Well-Tolerated in DMD Boys, Phase 1 Trial Results Show

News

Promising safety and tolerability data from a Phase 1 trial on suvodirsen (WVE-210201), Wave Life Sciences‘ investigational exon-skipping therapy for boys with Duchenne muscular dystrophy (DMD), opens the door for the launch ... Read more

April 17, 2019July 8, 2019

Pamrevlumab, Potential Treatment for Fibrosis in DMD Patients, Named Orphan Drug by FDA

News

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to pamrevlumab (FG-3019), FibroGen‘s potential anti-fibrosis treatment for Duchenne muscular dystrophy (DMD) patients. Pamrevlumab is a human monoclonal antibody that blocks the ... Read more

April 5, 2019June 21, 2019

Casimersen Shows Promising Phase 3 Results for DMD, May Open Door for FDA New Drug Application

News

Sarepta Therapeutics‘ casimersen (SRP-4045), one of the company’s investigational exon-skipping therapies for Duchenne muscular dystrophy (DMD), showed promising results in an interim analysis of an ongoing Phase 3 clinical trial. These positive ... Read more

March 15, 2019March 29, 2019

Vamorolone Safely Aids Skeletal and Heart Muscle, Animal Study Finds

News

Vamorolone, an experimental treatment for Duchenne muscular dystrophy (DMD), combines the benefits of two existing therapies — prednisone and eplerenone — on heart and muscle health, but with less detrimental side effects, ... Read more

November 19, 2018November 19, 2018

Catabasis, UT Southwestern Collaborate to Explore Effect of Edasalonexent on Heart Function in Muscular Dystrophies

News

Catabasis Pharmaceuticals will collaborate with the University of Texas Southwestern to investigate potential benefits of edasalonexent on heart function in Duchenne and Becker muscular dystrophies. After the promising results of ... Read more

October 15, 2018October 15, 2018

GsMTx4 a Promising ‘Out-of-the-Box’ Therapy for Advanced DMD, Mouse Study Shows

News

A new investigational therapy called GsMTx4, developed by researchers at the University of Buffalo and based on a molecule found in tarantula venom, is able to prevent the loss of muscle ... Read more

Posts by: Joana Carvalho

Joana Carvalho

Santhera Collaborating With Swiss Researchers to Develop New Gene Therapy for LAMA2 MD

PPMD Awards $100K for Immune Cell Profiling Research for Duchenne and Becker at UCLA

Lab Test Allows Prenatal Diagnosis of One Type of Congenital MD, Study Finds

Wave’s Exon 51-Skipping Therapy Safe, Well-Tolerated in DMD Boys, Phase 1 Trial Results Show

Pamrevlumab, Potential Treatment for Fibrosis in DMD Patients, Named Orphan Drug by FDA

Casimersen Shows Promising Phase 3 Results for DMD, May Open Door for FDA New Drug Application

Vamorolone Safely Aids Skeletal and Heart Muscle, Animal Study Finds

Catabasis, UT Southwestern Collaborate to Explore Effect of Edasalonexent on Heart Function in Muscular Dystrophies

GsMTx4 a Promising ‘Out-of-the-Box’ Therapy for Advanced DMD, Mouse Study Shows

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