0900 712 712
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0900 712 713
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0900 712 712
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0900 712 713
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University Children’s Hospital Basel
T +41 61 704 12 12
F +41 61 704 12 13
One of our main research interests is to identify objective clinical endpoints or surrogate imaging markers that are useful in evaluating disease progression in muscular dystrophies and related neuromuscular disorders. In close collaboration with the Department of Radiological Physics of the University Basel (Prof. Oliver Bieri) we are developing and evaluating novel quantitative MRI sequences to distinguish healthy from fatty degenerated muscle tissue. These methods are clearly superior to clinical scores when assessing the effect of novel interventions and treatments on disease progression.
Our second area of
scientific interest includes clinical studies aimed to improve the energetic
situation of muscle cells in patients with neuromuscular diseases. A positive
effect on muscle metabolism of a treatment with metformin and L-arginine was
assessed in a pilot study on 5 patients with Duchenne muscular dystrophy. Larger placebo controlled clinical
trials using the same hypothesis of an amelioration of muscle metabolism were
conducted on patients with Duchenne muscular dystrophy and Becker muscular
dystrophy and were funded by Swiss National Science Foundation.
Post-polio syndrome (PPS) is a condition that affects many polio
patients years after recovery from an initial acute attack of the poliomyelitis
virus. Most often patients start to experience slowly progressive muscle
weakness and fatigue. We perform a randomised, placebo controlled trial to
assess the influence of an oral treatment with L-citrulline on energy
metabolism in muscles of post-polio patients and its effect on muscle function,
endurance and quality of life. This study is sponsored by the Thomi-Hopf-Foundation.
Recruitment of patients has been terminated.
The examination of the peripheral nervous system
with ultrasonogaphy is an emerging non-invasive technique that became popular in the last few years. To distinguish a pathological swelling of the nerves from normal
aspects a comparison with normal values from a group of healthy children is
important. This investigator-initiated
study aims to establish normal values for cross sectional
areas of several upper and lower limb nerves and cervical roots in children and
adolescents from 2 to 18 years.
We are actually recruiting patients, please contact us.
At the university’s children hospital (UKBB),
the phase III, double-blind, randomized, placebo-controlled trial SIDEROS (sponsored
by Santhera®) aims to assess the efficacy of Raxone in slowing the rate of
respiratory function decline in Duchenne muscular dystrophy (DMD) patients
receiving concomitant glucocorticoids. Study participants will receive either
Raxone (900 mg/day; given as 2 tablets 3 times a day with meals) or placebo for
78 weeks (18 months). The primary endpoint of the trial is change from baseline
to week 78 in forced vital capacity % predicted (FVC%p). Patients completing
the trial will be offered the opportunity to enroll in an open label extension
study where all patients receive Raxone.
We are recruiting patients, please contact us.
In this study the effect of Tamoxifen is evaluated in boys with Duchenne muscular dystrophy. Using the mouse DMD model encouraging results have been shown concerning the muscle function during a treatment with tamoxifen. Patients will be recruited internationals in 9 centers (7 countries), the trial will be coordinated by the UKBB (sponsored by EU, SNF, patient organizations: Duchenne UK, NL and Monaco).
We are not yet recruiting patients. Please contact us for more information.
Approx. one billion people are suffering from migraine worldwide and yet, therapeutic options are still very limited. Research suggests that changes in energy metabolism could be part of migraine pathophysiology. Ketone bodies (KB) are endogenous alternative energy substrates. Our clinical trial assesses the efficacy and safety of KB supplements in 60-90 adult migraineurs (5-14 migraine days / months) at the University Hospital Basel. The total duration of the trial is approx. 6 months, consisting of 4 weeks baseline, 12 weeks intervention with KB powder or matched placebo and 8 weeks follow-up. The primary endpoint is the change in migraine days at the end of intervention compared to baseline. Additionally, changes in gene expression, fat-, protein- and glucose metabolism, inflammatory markers and quality of life will be examined.
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© UKBB, 2020