Patients with Fabry's disease, an X-linked lysosomal storage disorder, had a roughly 5% risk for major CV events, with the most common risk factors being hypertension and left ventricular hypertrophy, according to registry data.
Lysosomal Storage Disorders
FDA Denies Gaucher's Disease Drug; High Jewish Incidence
Gaucher's disease is the most common genetic disease affecting Ashkenazi Jewish people (Eastern, Central and Northern European ancestry), with a carrier frequency of ten percent. Today however, a new drug formulated by Protalix BioTherapeutics, Inc. has received bad news from the FDA in a response stating that the company's test reporting needs to be more detailed and clear.
Three-Year Data From Phase 2 Trial Of Genzyme Gaucher Disease Oral Compound Suggest Sustained Or Further Improvement Across All Endpoints
Genzyme Corporation (NASDAQ: GENZ) announced three-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. Sustained or further improvements were observed across all endpoints, including bone disease, at the three-year timepoint. The results were presented for the first time this week at the Lysosomal Disease Network WORLD Symposium in Las Vegas, Nevada.
Protalix BioTherapeutics Presents Data On The Company's Fabry Program And Oral Enzyme Gaucher Program With Experts In The Field Of Lysosomal Disorders
Protalix BioTherapeutics, Inc. (NYSE-AMEX: PLX, TASE: PLX), announced that management presented data on the Company's preclinical Fabry program and oral enzyme Gaucher program with experts in the field of lysosomal disorders at a Company-sponsored medical meeting which was recently held in New York City.
Synageva BioPharma Corp And To-BBB To Collaborate On Brain Drug Delivery For Rare Diseases
Synageva BioPharma Corp. and to-BBB technologies BV, the Dutch drug brain delivery company, have entered into a research collaboration to evaluate the potential of transporting therapeutic proteins across the blood-brain barrier into the central nervous system (CNS).