[49, 50] The International Consensus Panel on the Management and Treatment of MPS I recommends laronidase if the patient meets one of the following criteria: age >2?years; age ��2 and expected to have an attenuated phenotype; age ��2?years and expected to have a severe phenotype with a developmental intelligence quotient of less than 70. Idursulfase is a recombinant human iduronate-2-sulphatase (rhI2S) approved MS-275
for the treatment of MPS II. In a Phase 2/3 study carried out in 96 patients with a mean age of 15?years (range 5 to 31?years), patients received placebo, idursulfase 0��5?mg/kg weekly, or idursulfase 0��5?mg/kg every other week (EOW) for 53?weeks after which the primary endpoint, a composite score composed of FVC and 6MWT, was measured (Table?2). Compared with placebo, the weekly group showed a significant improvement in the primary endpoint by 18��96?��?6��47 (P?=?0��0049) and EOW group by 12��86?��?6��17 (P?=?0��0416). Both treatment groups achieved superior Y-27632 supplier
results in the secondary outcomes as well, such as the liver and spleen size and uGAG, with more favourable effects seen in the weekly than EOW group. Most adverse events occurred between 4 and 12?weeks and were generally mild. A total of 94 patients who completed the trial continued onto an open-label extension study to receive idursulfase once weekly (Table?2). After 2?years of treatment, changes in %FVC were minimal; however, an increase in the absolute FVC (+0��31?��?0��06?L; P?<?0��05) from the baseline was noted. Throughout the study, sustained improvements were seen in the 6MWT at regular intervals, with the largest increase at 20?months (+42?��?10 metres; P?<?0��001). Findings from the Hunter Outcome Survey conducted in 124 patients aged younger than 6?years showed acceptable efficacy and tolerability for idursulfase given for approximately 1?year. A longer follow-up (36?months) of a 4-year-old patient with a severe phenotype revealed an amelioration of organomegaly and bone remodelling; however, cardiac and neurologic symptoms progressively worsened despite treatment. A number of ERT guidelines for the treatment of MPS II have been published. In Australia, ERT is recommended only if the patient has the attenuated disease and should be withdrawn when neuropathic involvement is identified or the long-term prognosis is unlikely to be influenced by ERT. On the other hand, Hunter Syndrome <a href="http://www.selleckchem.com/products/AZD2281
(Olaparib).html">Olaparib European Expert Council declares all MPS II patients eligible for a 12- to 18-month trial of ERT except when the disease is so advanced that ERT is not expected to provide any benefit or when other life-threatening conditions are present. Galsulfase is a recombinant human N-acetylgalactosamine 4-sulphatase (rhASB) approved for the treatment of MPS VI. It is initiated preferably early in the disease trajectory and continued until neuronopathy or any other serious health condition compromises its efficacy.