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
July 18, 2017August 10, 2017
Cerdelga a Viable Option to Treat Nonresponsive ERT Gaucher Symptoms, Case Study Suggests

Substrate reduction therapy (SRT) may help manage Gaucher disease symptoms that do not respond to first-line enzyme replacement therapies ... Read more
July 13, 2017July 13, 2017
Imaging Techniques Combo Helps Better Evaluate Bone Disease Risk in Gaucher Patients, Study Finds

Researchers found that combining a new, noninvasive technique called trabecular bone score (TBS) with bone mineral density techniques helps to ... Read more
July 11, 2017July 11, 2017
Recommendations on Genetic Counseling for Gaucher-Parkinson’s Link Are Needed, Researchers Argue

Although people with Gaucher disease have a higher risk of developing Parkinson’s disease later in life, the link between ... Read more
July 6, 2017
FDA, EMA Propose Exploring Innovative Solutions for Gaucher Disease Drug Development

Regulatory agencies in the U.S. and Europe have joined forces to develop a proposal with the aim to spur ... Read more
June 29, 2017
Hearing Loss in Gaucher Disease Type 1 Patient Improved with Cochlear Implants

A young boy with Gaucher disease type 1 and hearing loss improved his hearing after receiving cochlear implants in ... Read more
June 27, 2017June 27, 2017
Excessive Anger, Aggressiveness Are Behavioral Symptoms of Gaucher Disease Type 3, Study Finds

Abnormal behavioral patterns, including increased aggressiveness and anger, can be symptoms of Gaucher disease (GD) type 3 and also ... Read more
June 20, 2017June 20, 2017
Taliglucerase Alfa Therapy Seen as Safe and Effective for Children with Gaucher Disease Type 1, Review Finds

The plant–based enzyme Elelyso (taliglucerase alfa), developed by Protalix and Pfizer as an intravenous enzyme replacement therapy (ERT), is ... Read more

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

July 18, 2017August 10, 2017

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

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 ... Read more

July 13, 2017July 13, 2017

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

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 ... Read more

July 11, 2017July 11, 2017

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

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. ... Read more

July 6, 2017

FDA, EMA Propose Exploring Innovative Solutions for Gaucher Disease Drug Development

Regulatory agencies in the U.S. and Europe have joined forces to develop a proposal with the aim to spur innovative approaches in the development of new Gaucher disease treatments. The ... Read more

June 29, 2017

Hearing Loss in Gaucher Disease Type 1 Patient Improved with Cochlear Implants

A young boy with Gaucher disease type 1 and hearing loss improved his hearing after receiving cochlear implants in both ears. A cochlear implant is an electronic medical device ... Read more

June 27, 2017June 27, 2017

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

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 ... Read more

June 20, 2017June 20, 2017

Taliglucerase Alfa Therapy Seen as Safe and Effective for Children with Gaucher Disease Type 1, Review Finds

The plant–based enzyme Elelyso (taliglucerase alfa), developed by Protalix and Pfizer as an intravenous enzyme replacement therapy (ERT), is seen as effective and safe for the treatment of children ... Read more