Miglustat in adult and juvenile patients with Niemann-Pick disease type C: long-term data from a clinical trial.

A randomized, controlled trial of miglustat indicated that miglustat (Zavesca) stabilized neurological disease over 12 months in adult and juvenile patients with Niemann-Pick disease type C (NP-C). We report data from a non-controlled, open-label extension to this initial randomized trial. All patients completing the randomized trial were allowed to continue treatment in a 12-month, non-controlled open-label extension.

Long-term miglustat therapy in children with Niemann-Pick disease type C.

Niemann-Pick disease type C is a rare, genetic disease associated with impaired intracellular lipid trafficking and progressive neurological symptoms. Miglustat slowed disease progression in a 12-month randomized trial in juveniles and adults with Niemann-Pick disease type C, and in a parallel, noncontrolled study in affected children. Here, the authors report the open-label extension to the pediatric study.

Phase II trial of CoQ10 for ALS finds insufficient evidence to justify phase III.

Objective: Amyotrophic lateral sclerosis (ALS) is a devastating, and currently incurable, neuromuscular disease in which oxidative stress and mitochondrial impairment are contributing to neuronal loss. Coenzyme Q10 (CoQ10), an antioxidant and mitochondrial cofactor, has shown promise in ALS transgenic mice, and in clinical trials for neurodegenerative diseases other than ALS. Our aims were to choose between two high doses of CoQ10 for ALS, and to determine if it merits testing in a Phase III clinical trial.

Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study.

Background: Niemann-Pick type C disease (NPC) is an inherited neurodegenerative disorder characterised by an intracellular lipid-trafficking defect with secondary accumulation of glycosphingolipids. Miglustat, a small iminosugar, reversibly inhibits glucosylceramide synthase, which catalyses the first committed step of glycosphingolipid synthesis. Miglustat is able to cross the blood-brain barrier, and is thus a potential therapy for neurological diseases.

Phase II study of neoadjuvant androgen deprivation followed by external-beam radiotherapy with 9 months of androgen deprivation for intermediate- to high-risk localized prostate cancer.

Purpose: To evaluate the toxicity and efficacy of individualized neoadjuvant androgen deprivation (AD) to maximal response followed by external beam radiotherapy (RT) with continued AD for a total of 9 months in a prospective phase II trial.

Patients And Methods: One hundred twenty-three patients received a total of 9 months of flutamide and luprolide combined with RT. RT initiation was individualized to begin after maximum response to AD as assessed by monthly digital rectal examination and prostate-specific antigen (PSA).

Localization of neurovascular bundles on pelvic CT and evaluation of radiation dose to structures putatively involved in erectile dysfunction after prostate brachytherapy.

Purpose: To (a) locate neurovascular bundles (NVB) on pelvic CT and (b) retrospectively evaluate relationships between radiation dose to structures putatively involved in prostate brachytherapy-induced erectile dysfunction (ED) and incidence of postbrachytherapy ED.

Methods And Materials: (a) Right/left NVB were identified on nine prostate MRIs. Structures visible on MRI and CT were cross-referenced.