News

August 24, 2017

Cerdelga Trial Extension Confirms Improvements in Gaucher Type 1 Patients

News

Gaucher disease type 1 (GD1) patients who continued Cerdelga (eliglustat) treatment in an extension study confirmed general organ improvement. The results of a Phase 3 trial extension were reported in ... Read more

August 22, 2017

Gaucher Patients Have Mixed Views About Substrate Reduction Therapies, Study Finds

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People with Gaucher’s disease have mixed views about using oral substrate reduction therapies as an alternative to enzyme replacement therapies, a study reports. Some favor substrate reduction therapies, or SRTs, ... Read more

August 15, 2017

UK Clinical Trial Aims to Characterize Symptoms and Therapy Response in Gaucher Types 1 and 3

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An ongoing clinical trial in the United Kingdom aims to determine factors that may predict the severity of Gaucher disease (GD) in adults and children, along with patients’ response to therapy. It focuses particularly on how ... Read more

August 1, 2017

Newborn Screening Platform for Lysosomal Storage Diseases Granted CE Mark in Europe

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Baebis recently announced that Seeker, the company’s novel newborn screening laboratory platform designed to measure the activity of lysosomal enzymes from newborn dried blood specimens, has been granted a CE Mark ... Read more

July 27, 2017July 27, 2017

Poor Bone Health in Gaucher Disease May Be Unrelated to the Condition Itself, Study Reports

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Poor bone health in people with Gaucher disease is likely driven by processes independent of the condition, researchers at Argentina’s National University of La Plata report. They identified the prime suspects in ... Read more

July 25, 2017

Long-term Follow-up Needed to Assess Newborn Screening for Lysomal Storage Disorders

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The benefits of newborn screening for lysosomal storage disorders such as Gaucher disease were not directly apparent 15 months after Illinois launched statewide screening. Long-term follow-up is needed to assess ... Read more

July 20, 2017

Sanofi Genzyme Planning Next Round of Scholarships for Students with Lysosomal Storage Disorders

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Sanofi Genzyme established the Chart Your Own Course scholarship program back in 2013 to help students with lysosomal storage disorders pursue their dreams. Judges will select the 15 winners of this year’s fifth annual ... Read more

July 18, 2017August 10, 2017

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

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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

July 13, 2017July 13, 2017

Imaging Techniques Combo Helps Better Evaluate Bone Disease Risk in Gaucher Patients, Study Finds

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Researchers found that combining a new, noninvasive technique called trabecular bone score (TBS) with bone mineral density techniques helps to better identify Gaucher disease patients at increased risk for bone fractures. ... Read more

July 11, 2017July 11, 2017

Recommendations on Genetic Counseling for Gaucher-Parkinson’s Link Are Needed, Researchers Argue

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Although people with Gaucher disease have a higher risk of developing Parkinson’s disease later in life, the link between the two diseases is rarely addressed in a genetic counseling setting. But ... Read more