Posts by: Joana Carvalho

Joana Carvalho

Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.

January 25, 2021January 25, 2021

Cerezyme Effective Over 20 Years in Easing GD1 Symptoms, Data Show

News

When given over two decades, Cerezyme (imiglucerase) was effective at easing most symptoms of Gaucher disease type 1 (GD1), according to long-term data from an international patient registry. Importantly, these ... Read more

November 23, 2020November 23, 2020

AVR-RD-02 Gene Therapy Lowers GD Biomarkers in First Patient Dosed in Trial

News

A single infusion of AVR-RD-02, Avrobio’s experimental gene therapy for Gaucher disease type 1, was able to lower the levels of two disease biomarkers within three months in the first ... Read more

September 14, 2020September 14, 2020

Fatty Liver Common, Linked to Scarring, in Type 1 Gaucher, Study Finds

News

Liver steatosis, a condition characterized by the buildup of fatty tissue inside the liver, is common in adults with type 1 Gaucher disease and is strongly associated with metabolic changes linked ... Read more

August 24, 2020August 24, 2020

Centogene, Evotec Expand Collaboration to Develop New Gaucher Treatments

News

Centogene and Evotec have expanded their collaboration agreement to focus on the development of new treatments for Gaucher disease and other rare disorders caused by mutations in the GBA gene, the ... Read more

July 6, 2020July 6, 2020

Lysogene, Weizmann Institute Team Up on Gene Therapy for Neuronopathic Gaucher

News

Lysogene has entered into a collaborative research agreement with Yeda Research and Development, a commercial branch of the Weizmann Institute of Science, to develop a gene therapy for neuronopathic Gaucher ... Read more

June 29, 2020June 29, 2020

Arimoclomol Shows Potential to Aid Gaucher Patients in Phase 2 Trial

News

Arimoclomol, Orphazyme’s investigational therapy for Gaucher disease and other disorders, leads to clinically meaningful, dose-dependent reductions in liver and spleen size in patients with type 1 or 3 Gaucher, according to ... Read more

June 22, 2020June 22, 2020

Long-term Cerezyme Followed by Cerdelga Delayed Bone Loss in Gaucher Type 1 Case Study

News

Using a non-invasive high-resolution imaging method, physicians found that long-term treatment with Cerezyme (imiglucerase) followed by a switch to Cerdelga (eliglustat) seemed to prevent or delay bone loss in a ... Read more

June 1, 2020June 1, 2020

Liver Health Needs Constant Monitoring in Gaucher Patients, Study Says

News

Although standard therapies for Gaucher disease (GD) usually reduce liver damage, some forms of treatment — particularly enzyme replacement therapy (ERT) — seem to be linked to a higher incidence ... Read more

May 18, 2020May 18, 2020

RANK Pathway Components May Be Markers of Bone Disease in Gaucher, Study Says

News

Components of the RANK pathway — a biological pathway that acts as a bridge between the immune and skeletal systems — may be used as markers of bone disease progression ... Read more

May 11, 2020May 11, 2020

Cognitive Impairment, Depression May Predict Parkinsonism in Gaucher Patients, Study Says

News

Non-motor symptoms, such as cognitive impairments and depression, may be used as early predictors of parkinsonism in Gaucher disease patients on enzyme replacement therapy (ERT), according to a recent study. ... Read more