Posts by: Alice Melao

News

Persistent high levels of iron in patients with Gaucher disease who are receiving maintenance therapy are associated with residual Gaucher disease. This finding was reported in the study “Iron storage ... Read more

September 7, 2017September 7, 2017

Nerve Cell Damage Causes Pain in Gaucher Disease Type 1 Patients, Italian Study Suggests

News

Pain is a common symptom among patients with Gaucher disease (GD). Italian researchers say it’s caused by peripheral nerve cell damage and not only bone damage, as was previously thought. Their ... Read more

September 5, 2017

Three Newly Identify Compounds Show Promise for Types 2 and 3 Gaucher Disease

News

Researchers have identified three new compounds that can cross the blood-brain barrier and may represent a new therapeutic avenue for types 2 and 3 Gaucher disease — two forms of ... Read more

July 18, 2017August 10, 2017

Cerdelga a Viable Option to Treat Nonresponsive ERT Gaucher Symptoms, Case Study Suggests

News

Substrate reduction therapy (SRT) may help manage Gaucher disease symptoms that do not respond to first-line enzyme replacement therapies (ERT), according to a case report featured in the journal Molecular Genetics ... Read more

June 27, 2017June 27, 2017

Excessive Anger, Aggressiveness Are Behavioral Symptoms of Gaucher Disease Type 3, Study Finds

News

Abnormal behavioral patterns, including increased aggressiveness and anger, can be symptoms of Gaucher disease (GD) type 3 and also markers of disease progression, according to researchers. The study, “Previously unrecognized ... Read more

June 1, 2017

England’s National Health Service to Make Cerdelga Available as Gaucher Therapy

News

Cerdelga (eliglustat) will become the first oral treatment that England’s National Health Service (NHS) makes available to patients with Gaucher disease. At the moment, the only treatment the health service covers ... Read more

May 23, 2017May 23, 2017

Cerdelga Improves Gastrointestinal Symptoms in GD Patient, Korean Case Report Shows

News

Cerdelga (eliglustat tartrate) can be effective in treating gastrointestinal lesions in patients with Gaucher disease (GD), according to a clinical case published in BMC Medical Genetics. This finding supports the use ... Read more

April 27, 2017

PPMI Clinical Trial Seeks to Uncover Link Between GBA Mutations and Parkinson’s

News

The National Gaucher Foundation has endorsed a clinical study designed to identify biomarkers of Parkinson’s disease (PD), and to evaluate the connection between this disease and the occurrence of genetic ... Read more

March 2, 2017

Suppressing Inflammation-promoting Protein Could Lead to Gaucher Therapies, Study Reports

News

Suppressing a protein that promotes the inflammation associated with Gaucher and other lysosomal storage diseases could lead to new treatments for the conditions, researchers say. Scientists from the Cincinnati Children’s ... Read more

October 27, 2016October 27, 2016

Artificial Protein Could Be New Therapeutic Tool for Gaucher Disease

News

An artificial version of the protein progranulin could reverse most cellular defects found in Gaucher disease, according to study led by researchers at NYU Langone Medical Center. Gaucher disease (GD), a rare ... Read more