Posts by: Joana Carvalho, PhD

Joana Carvalho, PhD

Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.

September 15, 2020September 15, 2020

Sarepta Poised to Start Next Trial of SRP-9001 for Duchenne MD

News

Sarepta Therapeutics has completed a meeting with the Office of Tissues and Advanced Therapies (OTAT) that clarified regulatory requirements to start the company’s next clinical trial of SRP-9001, an ... Read more

August 11, 2020August 11, 2020

Patient Feedback Being Sought on Puldysa for Possible NHS Funding

News

The National Institute for Health and Care Excellence (NICE) is urging patient organizations to gather feedback from those with Duchenne muscular dystrophy (DMD) who received Santhera Pharmaceuticals‘ investigational treatment ... Read more

June 9, 2020June 15, 2020

SRP-9003 Gene Therapy for LGMD2E Shows Benefits After 1 Year

News

A single administration of the gene therapy SRP-9003 at a low dose continues to lead to significant improvements in functional measures in three children with limb girdle muscular dystrophy ... Read more

May 26, 2020May 26, 2020

Trial Supports ATL1102’s Safety in Improving Muscle Strength, Function in DMD Boys

News

A Phase 2 trial of ATL1102 met its primary goal of demonstrating favorable safety and tolerability in boys with Duchenne muscular dystrophy (DMD), final study results show. ATL1102, developed by Antisense Therapeutics, ... Read more

April 16, 2020April 16, 2020

FDA Grants Orphan Drug Designation to Epirium’s EB 002 for Duchenne and Becker MD

News

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Epirium Bio’s EB 002 ((+)-epicatechin) for the treatment of Duchenne and Becker muscular dystrophies. The designation supports ... Read more

April 14, 2020April 14, 2020

Capricor Hosts Call to Discuss Heart Complications of DMD, Its CAP-1002 Cell Therapy

News

Capricor Therapeutics will host a conference call and webcast with a cardiac expert to discuss heart complications associated with Duchenne muscular dystrophy (DMD) and will also provide an update ... Read more

February 28, 2020February 28, 2020

Phase 2b Trial of Fulcrum’s Oral FSHD Treatment, Losmapimod, Fully Enrolled

News

Fulcrum Therapeutics announced that its Phase 2b trial assessing the safety and efficacy of losmapimod, an investigational oral treatment for facioscapulohumeral muscular dystrophy (FSHD), is fully enrolled. The trial ... Read more

February 21, 2020February 21, 2020

Antimalarial Medicine Chloroquine Improves Muscle Strength in Animal Models of DM1, Study Finds

News

Treatment with chloroquine increased the levels and restored the activity of Muscleblind-like RNA-binding proteins, improving muscle function and strength in animal models of myotonic dystrophy type 1, a study ... Read more

February 4, 2020February 4, 2020

Gene Editing Restores Dystrophin in Pig Model of Duchenne and Patient Cells, Study Reports

News

The gene-editing tool CRISPR/Cas9 was used to correct defects in the DMD gene and restore dystrophin protein production, lengthening the lives of pigs in a model of Duchenne muscular dystrophy (DMD) and ... Read more

January 29, 2020January 29, 2020

Saphyr System to Aid GeneDx in Detecting Mutations Causing Various Muscular Dystrophies

News

GeneDx has chosen Bionano Genomics’ technology, called Saphyr System, to aid in developing tests to detect mutations in genes that are associated with different forms of muscular dystrophy and other disorders. Saphyr ... Read more

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