Gaucher Disease News and Research

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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

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

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

FDA requests additional data on CMC section of NDA for taliglucerase alfa from Protalix BioTherapeutics

Protalix BioTherapeutics, Inc. announced today that, in connection with the New Drug Application (NDA) filed by the Company in December 2009 for taliglucerase alfa for the treatment of Gaucher disease, the U.S. Food and Drug Administration (FDA) has requested additional data regarding the Chemistry, Manufacturing and Controls (CMC) section of the NDA.

2 Feb 2010

Amicus Therapeutics presents corporate outlook for 2010 at Annual J.P. Morgan Healthcare Conference

Amicus Therapeutics today outlined the Company's three key strategic priorities and presented a corporate outlook for 2010 at the 28th Annual J.P. Morgan Healthcare Conference.

12 Jan 2010

Shire submits REPLAGAL BLA with the FDA

Shire plc , the global specialty biopharmaceutical company, today announced that it has submitted a BLA with the FDA for REPLAGAL(R) (agalsidase alfa), its enzyme replacement therapy for Fabry disease. REPLAGAL first received marketing authorization in the European Union in 2001, and is approved for the treatment of Fabry disease in 45 countries.

22 Dec 2009

Shire submits a MAA for velaglucerase alfa in Europe

Shire plc, the global specialty biopharmaceutical company, today announced that it has submitted a MAA to the European Medicines Agency for velaglucerase alfa, the company's enzyme replacement therapy in development for the treatment of Type 1 Gaucher disease.

24 Nov 2009

Shire receives FDA Priority Review for velaglucerase alfa NDA

Shire plc, the global specialty biopharmaceutical company, today announced that the United States Food and Drug Administration (FDA) has granted Priority Review for the New Drug Application (NDA) for velaglucerase alfa, the company's enzyme replacement therapy in development for the treatment of Type 1 Gaucher disease.

4 Nov 2009

Shire announces third quarter 2009 financial results

Shire plc, the global specialty biopharmaceutical company, announces results for the three months to September 30, 2009.

31 Oct 2009

Genzyme commences patient enrollment in Genz-112638 phase 3 trials

Genzyme Corporation today announced that the company has begun enrollment in the first of two global, multi-center, phase 3 trials of Genz-112638, a potential new oral therapy for Gaucher disease type 1. The two multi-national, multi-center trials are being conducted to evaluate the safety and efficacy of the small molecule.

21 Oct 2009

Positive results from Protalix BioTherapeutics Phase III UPLYSO clinical trial

Protalix BioTherapeutics, Inc., announced today positive top-line results from the Company's pivotal Phase III clinical trial of UPLYSO (taliglucerase alfa, previously referred to as prGCD) in treatment naive patients diagnosed with Gaucher disease.

15 Oct 2009

Preliminary Phase 2 study results of Plicera drug released

Amicus Therapeutics today announced preliminary results from its Phase 2 randomized, open-label study to assess the safety, tolerability and preliminary efficacy of its investigational drug, Plicera(TM) (afegostat tartrate), in treatment-naive adult patients with type 1 Gaucher disease.

2 Oct 2009

Gaucher Disease News and Research

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

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

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

FDA requests additional data on CMC section of NDA for taliglucerase alfa from Protalix BioTherapeutics

Amicus Therapeutics presents corporate outlook for 2010 at Annual J.P. Morgan Healthcare Conference

Shire submits REPLAGAL BLA with the FDA

Shire submits a MAA for velaglucerase alfa in Europe

Shire receives FDA Priority Review for velaglucerase alfa NDA

Shire announces third quarter 2009 financial results

Genzyme commences patient enrollment in Genz-112638 phase 3 trials

Positive results from Protalix BioTherapeutics Phase III UPLYSO clinical trial

Preliminary Phase 2 study results of Plicera drug released

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