Posts by: Marisa Wexler MS

Health-related quality of life does not change substantially over time among people with pulmonary arterial hypertension (PAH) — though PAH patients who experience more substantial disease progression tend to report ... Read more

September 20, 2021September 20, 2021

Vumerity Recommended for EU Approval as RRMS Treatment

Multiple Sclerosis News Today

A committee of the European Medicines Agency is recommending that Vumerity (diroximel fumarate) be approved as an oral treatment for adults with relapsing-remitting multiple sclerosis (RRMS) in the European Union. The ... Read more

September 17, 2021September 17, 2021

Actelion, Owkin Partner on Machine Learning to Improve Clinical Trials

Pulmonary Hypertension News

A joint effort between the startup Owkin and Actelion Pharmaceuticals, one of the Janssen companies, will seek to develop machine learning techniques that could improve how clinical trials are designed, and ... Read more

September 17, 2021September 17, 2021

LeaderMed, OPKO to Develop Treatments in Asia

Hemophilia News Today

Two pharmaceutical companies, LeaderMed Health Group and OPKO Health, are teaming up to develop, manufacture, and commercialize two experimental medications in China and other locations in Asia. One of the ... Read more

September 17, 2021September 17, 2021

Patients on Anti-CD20 Therapies Urged to Get COVID-19 Vaccine

COVID-19 News, Multiple Sclerosis News Today

While people with multiple sclerosis (MS) taking anti-CD20 therapies do not mount a robust antibody response after getting vaccinated against COVID-19, the vaccines do strongly activate other parts of the ... Read more

September 17, 2021September 17, 2021

Adults With CF Can Connect at BreatheCon

Cystic Fibrosis News Today

A two-day virtual event called BreatheCon will offer adults with cystic fibrosis (CF) an opportunity to connect and share their experiences through open and honest dialogue. Registration for the Sept. ... Read more

September 17, 2021September 17, 2021

Rim Lesions, Suggesting Chronic Inflammation, May Be Common

Multiple Sclerosis News Today

An imaging feature called a rim lesion is found in about 4 out of every 10 people with multiple sclerosis (MS), according to a review study. These rims, thought to ... Read more

September 16, 2021September 16, 2021

NIH Renews Grant to Research Mechanisms of COPD and IPF

COPD News Today

A $12.8 million five-year renewal grant from the National Institutes of Health (NIH) will fund research that seeks to better understand the molecular mechanisms that drive chronic obstructive pulmonary disease ... Read more

September 16, 2021September 16, 2021

BBP-418, Potential LGMD2i Therapy in Phase 2 Trial, on FDA Fast Track

Muscular Dystrophy News

BBP-418, an investigational disease-modifying medication that aims to improve muscle strength and function in people with limb-girdle muscular dystrophy type 2i (LGMD2i), has been given fast track designation by the U.S. ... Read more

September 16, 2021September 16, 2021

FDA Lifts Hold, Allows Phase 2 Trial of KVD824 for HAE to Open

Angioedema News

The U.S. Food and Drug Administration (FDA) has lifted the hold it had placed on KalVista Pharmaceuticals’ Phase 2 KOMPLETE trial of KVD824 as a treatment to prevent swelling attacks in people ... Read more

September 16, 2021September 16, 2021

Class of Molecules May Offer Opioid Alternative for Treating Pain

Multiple Sclerosis News Today

A class of molecules called positive allosteric modulators, or PAMs, may be useful in treating pain caused by nerve damage — a common symptom of multiple sclerosis (MS) — according ... Read more

September 15, 2021September 15, 2021

IRSF Joins Standard of Excellence Program

Rett Syndrome News

The International Rett Syndrome Foundation (IRSF), an organization focused on accelerating research to treat and cure Rett syndrome, has been appointed to the National Health Council (NHC) Standard of Excellence ... Read more

September 15, 2021September 15, 2021

New USP8 Gene Mutation Found in Pituitary Tumor of Cushing’s Patient

Cushing's Disease News

A newly discovered mutation in a gene called USP8 may contribute to the development of pituitary tumors, which are the underlying cause Cushing’s disease, researchers reported, describing the mutation. Their work ... Read more

September 15, 2021September 15, 2021

Low-alcohol Red Wine Reduced PAH Symptoms in Rats

Pulmonary Hypertension News

Regularly consuming low-alcohol red wine reduced signs of pulmonary arterial hypertension (PAH) in a rat model of the disease, a study shows. “The results of this study suggest that chronic ... Read more

September 15, 2021September 15, 2021

BeneFIX Again Preventive Therapy for Children With Hemophilia B

Hemophilia News Today

The U.S. Food and Drug Administration (FDA) has restored its approval of BeneFIX as a routine prophylactic, or preventive, treatment for bleeds in children and adolescents under 16 with hemophilia B. ... Read more