Good Start Genetics is expanding VeriYou, its physician-ordered carrier screening tests, to include screening for Gaucher disease and 18 other inherited genetic disorders that are especially prevalent among Ashkenazi Jews, or those of Eastern European origin. The expansion is being led by a partnership signed last November between Good…
The sales of Elelyso for the treatment of Gaucher disease have increased significantly so far this year, Protalix BioTherapeutics announced in a press release. Protalix BioTherapeutics is an Israel-based pharmaceutical company that develops plant cell-based medicines. Elelyso (taliglucerase alfa), a plant-based enzyme, has been widely used as an intravenous…
The Assistance Fund is now providing financial assistance to help patients living with Gaucher disease (GD). The new fund will provide GD patients and families with financial aid for copayments, coinsurance, deductibles, health insurance premiums and incidental medical expenses related to their disease. “Gaucher disease is a rare and complex…
A new screening device called the Seeker System, which detects lysosomal storage disorders (LSDs) like Gaucher disease in newborns, has received de novo clearance from the U.S. Food and Drug Administration (FDA). The de novo classification offers an alternative pathway to classify novel types of medical devices of…
The Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the granting of a marketing authorization for Yargesa (miglustat), for the treatment of adults with mild-to-moderate type 1 Gaucher disease. Yargesa is a generic of Zavesca, which has been authorized in the European…
Protalix BioTherapeutics has announced that Fundação Oswaldo Cruz (Fiocruz), an arm of the Brazilian Ministry of Health, has issued a letter of intent to purchase significant quantities of Uplyso (alfataliglicerase) to treat people with Gaucher disease in Brazil. Known as Elelyso (taliglucerase alfa) outside of Latin America, the treatment was first…
Protalix BioTherapeutics, a biopharma specializing in the development of recombinant therapeutic proteins, announced that the Brazilian National Health Surveillance Agency, known as ANVISA, has granted regulatory approval for Uplyso (alfataliglicerase) to treat Gaucher disease in children. ANVISA approved Uplyso in March 2013 for long-term enzyme replacement therapy (ERT)…
Global Genes, a global advocacy nonprofit working for patients and families of rare and genetic diseases, will help hundreds of patient advocates worldwide, including those for Gaucher disease, with travel stipends up to $100,000 so that they can attend this year’s 5th Annual RARE Patient Advocacy Summit. The…
Researchers at the Cairo University Pediatric Hospital, in Egypt and at the Baylor Research Institute in Dallas conducted a study on Egyptian patients with the genetic Gaucher disease (GD) type 3 and found that those patients have clinical outcomes that are very different from type 3 patients in other countries. The study, “…
Researchers at the Academic Medical Center Amsterdam in the Netherlands conducted a study with the aim of providing insight on the pathophysiological implications of iron accumulation in patients with Gaucher disease (GD). The study, “Hyperferritinemia and iron metabolism in Gaucher disease: Potential pathophysiological implications,” published in…