Gaucher's Disease News and Research

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Gaucher disease is an inherited metabolic disorder in which harmful quantities of a fatty substance called glucocerebroside accumulate in the spleen, liver, lungs, bone marrow, and sometimes in the brain.

VPRIV receives positive opinion from CHMP for treatment of type 1 Gaucher disease

Shire plc, the global specialty biopharmaceutical company, announced today that the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion on the marketing authorisation for VPRIV (velaglucerase alfa), the company's enzyme replacement therapy for the treatment of type 1 Gaucher disease.

28 Jun 2010

Genzyme signs consent decree of permanent injunction, agrees to disgorgement

Genzyme Corp. has signed a consent decree agreeing to correct manufacturing quality violations at its Allston, Mass., manufacturing facility and will turn over to the federal government $175 million in unlawful profits from the sale of products that were made at the plant, the U.S. Food and Drug Administration announced today.

25 May 2010

OnePath Gaucher Application available for patients with Type 1 Gaucher disease

Shire plc, the global specialty biopharmaceutical company, today announced the availability of the OnePath Gaucher Application. The first and only application developed specifically for patients in the U.S. with Type 1 Gaucher disease, it is free and ready for download at the iTunes Application store.

24 May 2010

Genzyme to initiate $2 billion stock buyback

Genzyme Corp. today announced that its Board of Directors has voted to pursue several actions to increase shareholder value. The company will initiate a $2 billion stock buyback, under which $1 billion of stock will be repurchased in the near term and financed with debt. The additional $1 billion of stock will be repurchased during the next 12 months.

7 May 2010

Researchers present positive results of Phase II clinical trial of eliglustat tartrate

Gaucher disease, a rare enzyme deficiency disorder, is one of many conditions with few approved treatment options for patients. In a study published online today in Blood, the journal of the American Society of Hematology, researchers present positive results of a Phase II clinical trial of eliglustat tartrate, an oral therapy in development to treat Gaucher disease.

4 May 2010

Protalix BioTherapeutics submits validation data for taliglucerase alfa manufacturing process to the FDA

Protalix BioTherapeutics, Inc., announced today it has submitted validation data regarding the Company's manufacturing process for taliglucerase alfa to the U.S. Food and Drug Administration (FDA). The Company made the submission in response to a request from the FDA for validation data from the Company's upgraded manufacturing facility. The FDA is currently reviewing the Company's New Drug Application (NDA) for taliglucerase alfa for the treatment of Gaucher disease.

27 Apr 2010

Enobia Pharma unveils findings from first HPP self-reported patient survey at ACMG 2010

Enobia Pharma Inc. unveiled findings from the first hypophosphatasia self-reported patient survey intended to evaluate the burden of illness associated with HPP at the 2010 American College of Medical Genetics Annual Clinical Genetics Meeting.

30 Mar 2010

Positive data from Shire's Phase III trial of TKT-034 in Type 1 Gaucher disease patients who switched to VPRIV

Shire plc, the global specialty biopharmaceutical company, today presented positive data from a Phase III clinical trial (TKT-034) designed to evaluate the safety of switching to VPRIV (velaglucerase alfa for injection), from imiglucerase, as well as an interim analysis of safety data from an ongoing multicenter open-label treatment protocol (HGT-GCB-058) implemented to provide VPRIV to patients affected by the continuing shortage of imiglucerase.

26 Mar 2010

Pathway Genomics' personal Genetic Health Report now available

Pathway Genomics Inc., a U.S. based genetic testing company, announced the immediate availability of its personal Genetic Health Report. This customized and innovative personal genetic health report contains information on more than 70 health conditions, including pharmacogenetics (prescription medication response), propensity for complex disease, and carrier status (pre-pregnancy health).

17 Mar 2010

Shire receives marketing approval from FDA for VPRIV

Shire plc, the global specialty biopharmaceutical company, announced today that the U.S. Food and Drug Administration has granted marketing approval for VPRIV, a human cell line derived enzyme replacement therapy (ERT) for the long-term treatment of Type 1 Gaucher disease in pediatric and adult patients.

27 Feb 2010

Gaucher disease: FDA approves VPRIV

The U.S. Food and Drug Administration has approved velaglucerase alfa for injection (VPRIV) to treat children and adults with a form of the rare genetic disorder Gaucher disease.

26 Feb 2010

Genetic Disorder Tracker introduced by Guidepoint Global

Guidepoint Global, LLC, a leading primary research firm, today announced the launch of their Genetic Disorder Tracker, a monthly longitudinal data release that captures commercial trends, on a worldwide basis, associated with the treatment of eight rare genetic disorders: Gaucher's disease, Fabry's disease, PKU, MPS I (Hurler Syndrome), MPS II (Hunter Syndrome), MPS VI (Maroteaux-Lamy Syndrome), Niemann-Pick disease and Pompe disease.

25 Feb 2010

Shire reports total revenues of $3,008 million for 2009

Shire plc the global specialty biopharmaceutical company, announces results for the year to December 31, 2009.

22 Feb 2010

SRI International establishes new Neurodegenerative Diseases Research Program

SRI International, an independent nonprofit research and development institute, announced today that a new Neurodegenerative Diseases Research Program has been established within its Center for Health Sciences. This new SRI group of neuroscientists and researchers working at the cutting edge of science brings $6.2 million of funding from the National Institutes of Health (NIH), including the National Institute of Neurological Disorders and Stroke (NINDS), the National Institute of Environmental Health Sciences (NIEHS), and National Institute of General Medical Sciences (NIGMS).

19 Feb 2010

Genzyme announces fourth-quarter and full-year 2009 financial results

Genzyme Corp., a diversified, global biotechnology company, today announced fourth-quarter and full year 2009 financial results and provided 2010 guidance that reflects growth across its businesses and a focus on strengthening core areas of the company.

18 Feb 2010

Genzyme announces 2-year data from eliglustat tartrate Phase 2 trial for Gaucher disease

Genzyme Corporation announced today two-year follow-up data from patients enrolled in the phase 2 clinical trial for its investigational oral therapy for Gaucher disease type 1 known as eliglustat tartrate (formerly Genz-112638). Continued improvements were observed across all endpoints, including bone disease, at the two-year timepoint, compared with baseline. The two-year results were presented for the first time today at the Lysosomal Disease Network WORLD Symposium in Miami, Fla.

12 Feb 2010

Positive results from Shire's velaglucerase alfa Phase III study for Type 1 Gaucher disease

Shire plc, the global specialty biopharmaceutical company, today presented positive results from its first Phase III study (TKT 032) evaluating safety and efficacy of velaglucerase alfa, its investigational enzyme replacement therapy for the treatment of Type 1 Gaucher disease. The data were presented in an oral presentation at the Lysosomal Disease Network (LDN) World Symposium in Miami, Florida.

12 Feb 2010

Additional data from Phase III clinical trial of taliglucerase alfa in patients with Gaucher disease presented

Protalix BioTherapeutics, Inc., announced today that additional data from the Company's pivotal Phase III clinical trial of taliglucerase alfa in patients with Gaucher disease was presented at the Annual Meeting of the Lysosomal Disease Network: WORLD Symposium 2010 in Miami, Florida, during an oral session titled, "Novel Enzyme Replacement Therapy for Gaucher Disease: Phase III Pivotal Clinical Trial with Plant Cell Expressed Recombinant Glucocerebrosidase (prGCD) - taliglucerase alfa."

12 Feb 2010

WORLD Symposium: Protalix Biotherapeutics to present data on taliglucerase alfa Phase III Gaucher disease trial

Protalix Biotherapeutics, Inc. today announced that data from its pivotal Phase III trial of taliglucerase alfa in patients with Gaucher disease will be presented at the Annual Meeting of the Lysosomal Disease Network: WORLD Symposium 2010, February 10-12, 2010 in Miami, Florida.

5 Feb 2010

Shire's clinical study results for treatment of Gaucher disease to be presented at LDN World Symposium

Shire plc, the global specialty biopharmaceutical company, today announced that data from one of three Phase III clinical trials for velaglucerase alfa, the company's enzyme replacement therapy (ERT) in development for the treatment of Type 1 Gaucher disease, will be presented at the Lysosomal Disease Network (LDN) World Symposium, February 10-12, 2010 in Miami, Florida.

4 Feb 2010

Gaucher's Disease News and Research

Further Reading

VPRIV receives positive opinion from CHMP for treatment of type 1 Gaucher disease

Genzyme signs consent decree of permanent injunction, agrees to disgorgement

OnePath Gaucher Application available for patients with Type 1 Gaucher disease

Genzyme to initiate $2 billion stock buyback

Researchers present positive results of Phase II clinical trial of eliglustat tartrate

Protalix BioTherapeutics submits validation data for taliglucerase alfa manufacturing process to the FDA

Enobia Pharma unveils findings from first HPP self-reported patient survey at ACMG 2010

Positive data from Shire's Phase III trial of TKT-034 in Type 1 Gaucher disease patients who switched to VPRIV

Pathway Genomics' personal Genetic Health Report now available

Shire receives marketing approval from FDA for VPRIV

Gaucher disease: FDA approves VPRIV

Genetic Disorder Tracker introduced by Guidepoint Global

Shire reports total revenues of $3,008 million for 2009

SRI International establishes new Neurodegenerative Diseases Research Program

Genzyme announces fourth-quarter and full-year 2009 financial results

Genzyme announces 2-year data from eliglustat tartrate Phase 2 trial for Gaucher disease

Positive results from Shire's velaglucerase alfa Phase III study for Type 1 Gaucher disease

Additional data from Phase III clinical trial of taliglucerase alfa in patients with Gaucher disease presented

WORLD Symposium: Protalix Biotherapeutics to present data on taliglucerase alfa Phase III Gaucher disease trial

Shire's clinical study results for treatment of Gaucher disease to be presented at LDN World Symposium

Achieving and maintaining ISO 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

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