September 21, 2017
High Levels of Iron During Therapy Linked to Residual Gaucher Disease, Study Finds

Persistent high levels of iron in patients with Gaucher disease who are receiving maintenance therapy are associated with residual ... Read more
September 19, 2017
Gaucher Disease Guidance Could Help Non-specialists Diagnosis It Better, Study Reports

Doctors who are not Gaucher disease experts should have straightforward and easy-to-follow diagnosis guidance so patients can be treated ... Read more
September 7, 2017September 7, 2017
Nerve Cell Damage Causes Pain in Gaucher Disease Type 1 Patients, Italian Study Suggests

Pain is a common symptom among patients with Gaucher disease (GD). Italian researchers say it’s caused by peripheral nerve cell ... Read more
September 5, 2017
Three Newly Identify Compounds Show Promise for Types 2 and 3 Gaucher Disease

Researchers have identified three new compounds that can cross the blood-brain barrier and may represent a new therapeutic avenue ... Read more
August 31, 2017
Accordant’s Gaucher Program Receives NCQA Disease Management Accreditation

Accordant’s health programs, including its Gaucher disease program, have received a special Disease Management (DM) Accreditation from the National ... Read more
August 24, 2017
Cerdelga Trial Extension Confirms Improvements in Gaucher Type 1 Patients

Gaucher disease type 1 (GD1) patients who continued Cerdelga (eliglustat) treatment in an extension study confirmed general organ improvement. ... Read more
August 22, 2017
Gaucher Patients Have Mixed Views About Substrate Reduction Therapies, Study Finds

People with Gaucher’s disease have mixed views about using oral substrate reduction therapies as an alternative to enzyme replacement ... Read more
August 15, 2017
UK Clinical Trial Aims to Characterize Symptoms and Therapy Response in Gaucher Types 1 and 3

An ongoing clinical trial in the United Kingdom aims to determine factors that may predict the severity of Gaucher disease (GD) in ... Read more
August 1, 2017
Newborn Screening Platform for Lysosomal Storage Diseases Granted CE Mark in Europe

Baebis recently announced that Seeker, the company’s novel newborn screening laboratory platform designed to measure the activity of ... Read more
July 27, 2017July 27, 2017
Poor Bone Health in Gaucher Disease May Be Unrelated to the Condition Itself, Study Reports

Poor bone health in people with Gaucher disease is likely driven by processes independent of the condition, researchers at ... Read more
July 25, 2017
Long-term Follow-up Needed to Assess Newborn Screening for Lysomal Storage Disorders

The benefits of newborn screening for lysosomal storage disorders such as Gaucher disease were not directly apparent 15 months ... Read more
July 20, 2017
Sanofi Genzyme Planning Next Round of Scholarships for Students with Lysosomal Storage Disorders

Sanofi Genzyme established the Chart Your Own Course scholarship program back in 2013 to help students with lysosomal storage disorders pursue their dreams. ... Read more

High Levels of Iron During Therapy Linked to Residual Gaucher Disease, Study Finds

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 ... Read more

September 19, 2017

Gaucher Disease Guidance Could Help Non-specialists Diagnosis It Better, Study Reports

Doctors who are not Gaucher disease experts should have straightforward and easy-to-follow diagnosis guidance so patients can be treated earlier, according to a study reporting that it can take ... Read more

September 7, 2017September 7, 2017

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

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. ... Read more

September 5, 2017

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

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 ... Read more

August 31, 2017

Accordant’s Gaucher Program Receives NCQA Disease Management Accreditation

Accordant’s health programs, including its Gaucher disease program, have received a special Disease Management (DM) Accreditation from the National Committee for Quality Assurance (NCQA). NCQA is a non-profit organization ... Read more

August 24, 2017

Cerdelga Trial Extension Confirms Improvements in Gaucher Type 1 Patients

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 ... Read more

August 22, 2017

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

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 ... Read more

August 15, 2017

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

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 ... Read more

August 1, 2017

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

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 ... Read more

July 27, 2017July 27, 2017

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

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 ... Read more

July 25, 2017

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

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 ... Read more

July 20, 2017

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

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 ... Read more