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- Lundin, Anders, et al.
(författare)
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Efficacy and Safety of the Dopaminergic Stabilizer Pridopidine (ACR16) in Patients With Huntington's Disease
- 2010
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Ingår i: Clinical neuropharmacology. - 0362-5664 .- 1537-162X. ; 33:5, s. 260-264
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Tidskriftsartikel (refereegranskat)abstract
- Objectives: To evaluate the efficacy and safety of the dopaminergic stabilizer pridopidine (ACR16) in patients with Huntington's disease (HD). Methods: In a randomized, double-blind, placebo-controlled, 4-week trial, patients with HD received pridopidine (50 mg/d, n = 28) or placebo (n = 30). The primary outcome measure was the change from baseline in weighted cognitive score, assessed by cognitive tests (Symbol Digit Modalities, verbal fluency, and Stroop tests). Secondary outcome measures included changes in the Unified Huntington's Disease Rating Scale, Hospital Anxiety and Depression Scale, Leeds Sleep Evaluation Questionnaire, Reitan Trail-Making Test A, and Clinical Global Impression of Change. Safety assessments were also performed. Results: There was no significant difference between pridopidine and placebo in the change from baseline of the weighted cognitive score. However, secondary measures such as affective symptoms showed trends toward improvement, and there was significant improvement in voluntary motor symptoms compared with placebo (P<0.05). Pridopidine was well tolerated, with a safety profile similar to placebo. Conclusions: Pridopidine shows promise as a treatment for some of the symptoms of HD. In this small-scale study, the most notable effect was improvement in voluntary motor symptoms. Larger, longer-term trials are warranted.
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| 2. |
- Malm, Gunilla, et al.
(författare)
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Mucopolysaccharidoses in the Scandinavian countries: incidence and prevalence.
- 2008
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Ingår i: Acta paediatrica (Oslo, Norway : 1992). - : Wiley. - 1651-2227 .- 0803-5253. ; 97:11, s. 1577-81
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Tidskriftsartikel (refereegranskat)abstract
- AIM: The aim of this study was to estimate the incidence and prevalence of mucopolysaccharidoses (MPS disorders) in Scandinavia. METHODS: The retrospective period used for the incidence study covered the period from 1975 to 2004 in Sweden and Denmark and from 1979 to 2004 in Norway. Prevalence was derived from the number of MPS patients alive as of December 31, 2007. RESULTS: The incidence of all MPS disorders was 1.75 cases in Sweden, 3.08 cases in Norway and 1.77 cases in Denmark per 100 000 newborns. The incidence of MPS I was the most common in all three countries, with 0.67, 1.85 and 0.54 cases per 100 000 newborns, respectively; for MPS II, numbers were 0.27, 0.13 and 0.27, respectively. For patients with other MPS disorders the incidence varied widely. The prevalence for all MPS disorders was 4.24, 7.06 and 6.03 per 1 000 000 inhabitants in Sweden, Norway and Denmark, respectively. CONCLUSION: From three Scandinavian countries the incidence of MPS disorders is retrospectively evaluated for 25 years in Norway and 30 years in Sweden and Denmark. Incidence and prevalence studies of lysosomal disorders are prerequisites for cost benefit calculations in the face of newly developed and expensive therapies in the future.
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