Posts by: Joana Carvalho

Joana Carvalho

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.

February 16, 2021February 16, 2021

Deleting Mss51 Gene Reduces Fatigue, Increases Endurance in DMD Mice

News

By deleting the Mss51 gene, scientists were able to reduce fatigue, increase endurance, and ease mitochondrial impairment in a mouse model of Duchenne muscular dystrophy (DMD). Mss51 is a gene ... Read more

January 21, 2021January 21, 2021

New CureDuchenne Initiative Provides Support, Education on COVID-19 Vaccination

News

CureDuchenne has launched a new initiative to prioritize the needs of the Duchenne muscular dystrophy (DMD) community during the COVID-19 vaccine rollout. The nonprofit, which supports patients with DMD, their caregivers, ... Read more

January 5, 2021January 5, 2021

Edasalonexent Helps to Preserve Heart and Bone Health, New Data Show

News

Edasalonexent, a potential oral therapy for Duchenne muscular dystrophy (DMD), can prevent the development of heart disease and help to preserve bone health, according to data from two studies in ... Read more

October 13, 2020October 13, 2020

Phase 3 Trial of Puldysa Stopped for Lack of Efficacy in Duchenne Patients

News

Santhera Pharmaceuticals has decided to stop SIDEROS, its Phase 3 trial assessing the effectiveness of Puldysa (idebenone) at delaying respiratory decline in patients with Duchenne muscular dystrophy (DMD) on a ... Read more

October 1, 2020October 1, 2020

Durable Safety and Motor Gains Evident in Trial of SRP-9001, DMD Gene Therapy

News

A single dose of SRP-9001, Sarepta Therapeutics’ investigational gene therapy for Duchenne muscular dystrophy (DMD), continues to be safe and to improve motor function in four boys treated two years ago, according to ... Read more

September 24, 2020March 15, 2021

Vamorolone Continues to Improve Motor Function in DMD Boys, Phase 2 Trials Show

News

The experimental therapy vamorolone can improve motor function significantly in boys with Duchenne muscular dystrophy (DMD), without stunting their growth and with fewer side effects compared to standard corticosteroids, according to 18-month data ... Read more

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 investigational ... 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 Puldysa ... 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 (LGMD) ... Read more