COVID-19 News

July 20, 2021July 20, 2021

Work in Progress on Challenges of Minority Representation in CF Trials

COVID-19 News, Cystic Fibrosis News Today

Proper representation in clinical trials has long been a challenge for minorities with rare diseases like cystic fibrosis (CF), risking — and often leading to — inequities in care and ... Read more

July 20, 2021July 20, 2021

Masitinib, Given Early in ALS Course, Extends Life, Trial Data Show

ALS News Today, COVID-19 News

Treatment with the investigational oral therapy masitinib can extend lifespan when given early in the course of amyotrophic lateral sclerosis (ALS), according to a new study. “These long-term survival data, ... Read more

July 20, 2021July 21, 2021

DMD Trial Testing Oral ASP0367 Recruiting Boys 8–16 in US

COVID-19 News, Muscular Dystrophy News

A Phase 1b clinical trial evaluating Astellas Pharma’s ASP0367 is recruiting boys with Duchenne muscular dystrophy (DMD), ages 8–16, at six U.S. sites. The ongoing trial (NCT04184882) is evaluating the safety and ... Read more

July 20, 2021July 20, 2021

Lungs ‘Primed’ for Severe COVID-19 in People With IPF, Like Disorders

COVID-19 News, Pulmonary Fibrosis News

Changes in gene activity and immune cell populations in the lungs of people with chronic lung diseases, such as idiopathic pulmonary fibrosis (IPF), may predispose these patients to severe COVID-19, ... Read more

July 19, 2021July 20, 2021

Early Phase 1 Data Support PHA121 as Oral HAE Prophylactic

Angioedema News, COVID-19 News

Multiple doses of PHA121 (PHA-022121), an investigational oral therapy for hereditary angioedema (HAE), were well-tolerated and rapidly reached therapeutic levels in healthy volunteers, according to data from a Phase 1 ... Read more

July 19, 2021July 19, 2021

‘Wearable Cyborg’ Helps Improve Walking Ability, Muscle Strength

COVID-19 News, SMA News Today

A wearable cybernetic leg significantly improved the gait and ability to perform walking exercises of people with neuromuscular disorders, such as spinal muscular atrophy (SMA), as compared to a conventional ... Read more

July 19, 2021July 19, 2021

Enrollment in Phase 3 Masitinib Trial Expected to Soon Resume in France

ALS News Today, COVID-19 News

AB Science’s proposed measures to ensure patient safety in clinical trials of its experimental oral therapy masitinib were deemed sufficient by the French National Agency for Medicines and Health Products ... Read more

July 19, 2021July 19, 2021

Automated Imaging Method Seen to Accurately Capture PF Severity

COVID-19 News, Pulmonary Fibrosis News

A new, automated computer image analysis technique was able to accurately assess and measure the severity of pulmonary fibrosis (PF) using patients’ lung tissue samples, a study reported. According to ... Read more

July 16, 2021July 16, 2021

Grant Supports Miniprotein Binder as Potential IPF Therapy

COVID-19 News, Pulmonary Fibrosis News

The Washington Research Foundation (WRF) has awarded a $250,000 grant to support the development of a novel miniprotein binder to help treat idiopathic pulmonary fibrosis (IPF). The grant was awarded ... Read more

July 16, 2021July 16, 2021

California Earmarks $8.4M for Parkinson’s Disease Registry

COVID-19 News, Parkinson's News Today

California has committed $8.4 million to the California Parkinson’s Disease Registry (CPDR) and efforts to expand it to other neurodegenerative diseases over the next four years. The Michael J. Fox ... Read more

July 15, 2021July 16, 2021

Study Will Test Using Calcitriol to Boost Frataxin

COVID-19 News, Friedreich's Ataxia News

A clinical trial will assess whether supplementation with calcitriol, the active form of vitamin D, can boost frataxin production in people with Friedreich’s ataxia (FA). The Biochemistry of Oxidative Stress ... Read more

July 15, 2021July 15, 2021

Anti-CD20 Antibodies Reduce Myelin Loss in MS Rats

COVID-19 News, Multiple Sclerosis News Today

Treatment with anti-CD20 antibodies reduced the loss of myelin and improved the survival of neurons in a rat model of multiple sclerosis (MS), a new study reports. The findings imply ... Read more

July 15, 2021July 15, 2021

Eli Lilly, Verge Partner to Identify and Test Potential ALS Therapies

ALS News Today, COVID-19 News

Verge Genomics and Eli Lilly have entered into a three-year collaboration to find and develop therapies for amyotrophic lateral sclerosis (ALS). Verge will focus on discovering and validating potential new therapeutic targets, ... Read more

July 14, 2021July 15, 2021

Stem Cell Transplant Successfully Treats Bone Marrow Porphyrias

COVID-19 News, Porphyria News

A blood stem cell transplant can successfully correct defects in the body’s ability to make heme — a molecule that helps transport oxygen through the bloodstream — in people with ... Read more

July 14, 2021July 14, 2021

Rett Researcher Discovers Molecular Mechanism of MECP2 Binding

COVID-19 News, Rett Syndrome News

The molecular mechanism by which the protein MeCP2 recognizes and binds to CA repeats — repetitive short sequences of DNA that are found throughout the genome — has been unraveled ... Read more