Posts by: Forest Ray PhD

Angioedema News

Japan’s National Health Insurance System (NHI) has approved the addition of Orladeyo (berotralstat), an oral preventive treatment for hereditary angioedema (HAE) attacks, to its drug price list. This means that ... Read more

April 15, 2021April 15, 2021

Scleroderma Foundation Awards 8 Grants to Advance Research Priorities

Scleroderma News

The Scleroderma Foundation has awarded eight research grants for projects seeking to advance personalized medicine, lab-grown skin, new therapeutic targets, and more, in the field of scleroderma research. “The proposals ... Read more

April 15, 2021April 15, 2021

Intense Rehabilitation Can Help People at Earlier Disease Stages, Study Finds

Parkinson's News Today

An intensive and sweeping rehabilitation program, given in a hospital, led to significant short-term improvements in the functional and cognitive abilities of people with mild-to-moderate Parkinson’s disease in a recent study. ... Read more

April 14, 2021April 14, 2021

Signifor Controlled Patient’s Cortisol Fluctuations

Cushing's Disease News

Cortisol fluctuations in a woman with Cushing’s disease were controlled successfully with Signifor (pasireotide LAR), possibly for the first time, according to a recent case report. The report, “Resolution of ... Read more

April 14, 2021April 14, 2021

Board Recommends All Anavex 2-73 Trials Proceed

Rett Syndrome News

An independent monitoring board has recommended that all three ongoing clinical trials of Anavex 2-73 (blarcamesine), an investigational treatment for Rett syndrome, continue without changes, based on a review of interim safety ... Read more

April 14, 2021April 14, 2021

Ionis Plans to Launch Clinical Trial This Year for ION582

Angelman Syndrome News

Ionis Pharmaceuticals intends to launch a clinical trial later this year to test ION582, a potential Angelman syndrome therapy. The Phase 1/2a trial, designed to enroll up to 50 patients, ... Read more

April 14, 2021April 14, 2021

How Mutated Parkin Protein Causes Form of Early Onset Parkinson’s Detailed

Parkinson's News Today

The parkin protein, whose mutated form is linked to certain cases of early onset Parkinson’s disease, protects neurons by lowering levels of potentially harmful oxidants, and by removing them from areas ... Read more

April 13, 2021April 13, 2021

Galectin‐10 Blood Levels Linked With Inflammation, Lung Remodeling in Study

Scleroderma News

Low levels of the galectin-10 protein (Gal-10) in scleroderma patients associate with inflammation and vascular changes in the lungs, leading to pulmonary arterial hypertension (PAH). This finding, from a recent study, ... Read more

April 13, 2021April 13, 2021

Study Shows Difficulty of Adapting Movements to Obstacles

Parkinson's News Today

People with Parkinson’s disease are less able to adapt their movements before crossing obstacles such as mounting a curb, according to a recent study. This type of adaptation, called locomotor ... Read more

April 12, 2021April 12, 2021

Life Quality Gains Evident With Newer Preventive HAE Therapies, Survey Finds

Angioedema News

Newer preventive therapies for hereditary angioedema (HAE) lead to a better quality of life and fewer attacks than on-demand treatments, according to a large patient survey study. The study, “Assessing ... Read more

April 12, 2021April 12, 2021

Weight Loss, Other GI Symptoms, Common in FAP, Italian Study Finds

FAP News Today

Gastrointestinal (GI) symptoms, particularly unintentional weight loss, occur frequently in people with hereditary transthyretin amyloidosis, also known as familial amyloid polyneuropathy (FAP), according to data from an Italian study. Indeed, ... Read more

April 12, 2021April 12, 2021

US Survey Seeks to Understand Effects of MS on Patient Community

Multiple Sclerosis News Today

People living with multiple sclerosis (MS) and their caregivers are invited to help the patient and healthcare communities better understand MS by participating online in the 9th annual MS in ... Read more

April 9, 2021April 9, 2021

Cardiac to Dental Problems Can Be Early Signal of Fragile X

Fragile X News Today

Physical health is also affected by fragile X syndrome, with problems evident in the heart, digestive system, respiratory tract and even the teeth years before the cause of patients’ intellectual difficulties ... Read more

April 9, 2021April 9, 2021

Recorlev Also Helps to Treat Diabetes in Cushing’s Syndrome, Trial Data Show

Cushing's Disease News

Recorlev (levoketoconazole), Strongbridge Biopharma’s oral investigational therapy for endogenous Cushing’s syndrome, led to sustained improvements in cortisol and in blood sugar control among patients with both Cushing’s and diabetes, a subgroup ... Read more

April 8, 2021April 8, 2021

Single, Low-dose Course of Rituximab Treats MuSK-MG, Pilot Study Suggests

Myasthenia Gravis News

A single, two-day and low-dose course of rituximab can effectively treat patients with muscle-specific tyrosine kinase myasthenia gravis (MuSK-MG) over at least six months, according to a pilot study. The ... Read more