Clinical trial for Fabry disease faces continuing hurdles

Silversides, Ann
November 2009
CMAJ: Canadian Medical Association Journal;11/24/2009, Vol. 181 Issue 11, pE251
Academic Journal
The article informs that the clinical trial for Fabry disease conducted by the Canadian Fabry Disease Initiative is facing problems because of the worldwide shortage of one of the enzyme replacement therapies. It is stated that the trial involves a comparison of the agalsidase beta (Fabrazyme) and agasidase alfa (Replegal), but the global shortage of agalsidase beta has created new challenges like reducing dose of Fabrazyme to patients and threatening the integrity of the post market trial.


Related Articles

  • Clinical trial shows safety and efficacy of agalsidase beta.  // Ophthalmology Times;10/15/2003, Vol. 28 Issue 20, p27 

    Focuses on the clinical trial for the safety and efficacy of agalsidase beta, an enzyme replacement therapy for the treatment of Fabry's disease. Prevention of reactions associated with the enzyme infusion; Assessment of the levels of globotriaosylceramide inclusions in the renal interstitial...

  • Drugging the God of Thunder.  // BioWorld Today;4/18/2011, Vol. 22 Issue 74, Special section p2 

    The article focuses on a study which discovered the enzyme thorase, which is capable of removing glutamate receptors from synapses.

  • Clinic Roundup.  // BioWorld Today;3/21/2011, Vol. 22 Issue 54, p5 

    This article announces the enrollment by Polaris Group of the first patient in a Phase II trial of ADI-PEG 20, its enzyme-based treatment for malignant mesothelioma.

  • Evaluation of prescribing trends and rationality of use of oral proteolytic enzymes. Shah, Saumil A.; Nerurkar, Rajan P. // Indian Journal of Pharmacology;May/Jun2013, Vol. 45 Issue 3, p309 

    The article discusses the trends of prescribing drugs and the use of oral proteolytic enzymes, such as serratiopeptidase and chymotrypsins, in India. It mentions that there are several expensive drugs being prescribed without verifying the efficacy in a scientific manner or randomized controlled...

  • Role of Hydroxamate-Based Histone Deacetylase Inhibitors (Hb-HDACIs) in the Treatment of Solid Malignancies. Grassadonia, Antonino; Cioffi, Pasquale; Simiele, Felice; Iezzi, Laura; Zilli, Marinella; Natoli, Clara // Cancers;Sep2013, Vol. 5 Issue 3, p919 

    Hydroxamate-based histone deacetylase inhibitors (Hb-HDACIs), such as vorinostat, belinostat and panobinostat, have been previously shown to have a wide range of activity in hematologic malignancies such as cutaneous T-cell lymphoma and multiple myeloma. Recent data show that they synergize with...

  • Oral maintenance clinical trial with miglustat for type I Gaucher disease: switch from or combination with intravenous enzyme replacement.  // Current Medical Literature: Lysosomal Storage Disease;2008, Vol. 8 Issue 1, p18 

    The article reports on the result of the medical research on oral maintenance clinical trial with miglustat for type I Gaucher disease. It states that 36 patients were assigned to treatment groups according to clinical severity, age, sex, and years on enzyme replacement therapy (ERT). It says...

  • Observational Data Under the Microscope. Kubick, Wayne R. // Applied Clinical Trials;Apr2009, Vol. 18 Issue 4, p34 

    The article discusses the importance of using longitudinal health care data in clinical research in the U.S. It says that the data can be used in providing early warnings and assessment of potential safety issues, exploring and verifying safety signals of the patients, and for better...

  • Clinical Trials and Medical Care: Defining the Therapeutic Misconception. Henderson, Gail E.; Churchill, Larry R.; Davis, Arlene M.; Easter, Michele M.; Grady, Christine; Joffe, Steven; Kass, Nancy; King, Nancy M. P.; Lidz, Charles W.; Miller, Franklin G.; Nelson, Daniel K.; Peppercorn, Jeffrey; Rothschild, Barbra Bluestone; Sankar, Pamela; Wilfond, Benjamin S.; Zimmer, Catherine R. // PLoS Medicine;Nov2007, Vol. 4 Issue 11, pe324 

    A key component of informed consent to participate in medical research includes understanding that research is not the same as treatment.

  • RisedronatE and ALendronate Intervention over Three Years (REALITY): minimal differences in fracture risk reduction. Curtis, J. R.; Westfall, A. O.; Cheng, H.; Saag, K. G.; Delzell, E. // Osteoporosis International;Jun2009, Vol. 20 Issue 6, p973 

    The comparative effectiveness of alendronate and risedronate has received limited evaluation. Among 19,063 new users of bisphosphonates, risedronate users had a higher relative rate of hip fracture compared to alendronate users, but the difference in absolute fracture rate was small. We conclude...


Read the Article


Sorry, but this item is not currently available from your library.

Try another library?
Sign out of this library

Other Topics