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A phase IIb, open-label study to assess the efficacy, safety, pharmacodynamics and pharmacokinetics of multiple subcutaneous doses of BMN 045 (previously known as PRO045) in subjects with Duchenne muscular dystrophy

Primary objective:To assess the efficacy of BMN 045 after 48 weeks treatment in ambulant subjects with Duchenne muscular dystrophy.Secondary objectives:To assess the safety and tolerability of BMN 045 after 48 weeks of treatment in all study…

An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy

Primary:The purpose of this study is to evaluate the safety, tolerability, orally administered LMI070 in patients with Type 1 SMA.pharmacokinetics (PK), pharmacodynamics (PD) and efficacy; and to estimate the Maximum Tolerated Dose (MTD) and optimal…

A Phase I/II, open-label, dose escalating with 48-week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of BMN 053 (previously knoen as PRO053) in subjects with Duchenne muscular dystrophy

Primary objective: To assess the efficacy of BMN 053 at recommended dosing regimen after 48 weeks treatment in ambulant subjects with Duchenne muscular dystrophy.Secondary objectives: - To assess the safety and tolerability of BMN 053 after single…

Multicenter, open-label, single arm study to evaluate long-term safety, tolerability, and effectiveness of 10mg/kg olesoxime in patients with SMA

Main objective: The main objective of this open-label, single arm study is to further characterize the safety, tolerability and effectiveness profile of olesoxime in SMAThe primary safety objective for this study is as follows:To evaluate the safety…

The effects of body weight support, guidance, and gait speed on the patterning of muscle activity in the Lokomat

a. To establish the effects of (1) body weight support (2) treadmill speed, and (3) level of movement guidance provided by the exoskeleton on the neuromuscular control of the lower extremities.b. To establish the interactions between the above…

A Phase 1, 2-part, Open Label Pharmacokinetic and Safety Study to Evaluate Dose Proportionality and Accumulation of Firdapse® (3,4-Diaminopyridine Phosphate) after Single and Multiple Oral Doses in Healthy Volunteers

Primary:To assess the safety and tolerability of Firdapse after single and multiple doses Secondary:Part 1 only: To determine the dose-related PK profile of single, escalating doses of Firdapse;Part 2 only: To assess the steady-state plasma PK and…

6 minutes assisted leg and arm cycling test: feasibility, validity, test-retest responsivity and responsivity in patients with neuromuscular and metabolic disorders

2.1 Primary Objectives: 1) To test the feasibility of the six minute assisted leg and arm cycling test in patients aged 6-18 years with Duchenne muscular dystrophy (DMD), Beckers muscular dystrophy (BMD), Limb Girdle muscular dystrophy (LGMD),…

An Open-Label, Multicenter, Extension Study to Evaluate the Long-Term Safety and Efficacy of Dexpramipexole (BIIB050) in Subjects With Amyotrophic Lateral Sclerosis

Primary:The primary objective of the study is to evaluate the long-term safety profile of dexpramipexole in subjects with ALS.Secondary:The secondary objective of this study is to evaluate the longterm efficacy of dexpramipexole in this study…

Botulinum toxin A intravesical injections in the treatment of Bladder Pain Syndrome/
Interstitial Cystitis.

To describe the course of symptoms of BPS/IC after intravesical injection of Botox®.

An evaluation of gait and patient experience of two newly developed stance-control knee joints for custom-made KAFO*s, a pilot study.

The aim of this study is to investigate whether the new joints for the sc-kevo's can be effectively operated by the patient, and whether a minimal hip extensor strength is needed to operate the joints succesfully. Secondly, the effect of the…

Determinants of success of a stance-control-KAFO in former
polio patients with quadriceps weakness.

The aim of the present study is two-fold: 1) To do a survey of patients who have been prescribed a SC-KAFO in the outpatient clinic and evaluate the indication of prescription, compliance, experience and motives for (non) use of the SC-KAFO. 2) To…

An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704

The objective of this extension study is to assess the long-term safety and efficacy of Myozyme treatment in patients with Late-Onset Pompe Disease who were previously treated under the placebo-controlled, double-blind study AGLU02704.

Improving the accuracy of determination of onset of neuromuscular block

The primary objective of the study is to determine if twitch response following rocuronium administration evoked by 1 Hz ST neuromuscular monitoring can be modeled to a similar degree of accuracy as previously published studies describe for 0.1 Hz (…

Analysis of ankle range of motion, ground reaction forces and walking ability during walking with the Foot-up orthosis.

The goal of this research is to discover if there is an improvement in the walking ability when someone walks with the Foot-up compared to the plastic AFO and walking without an AFO. The Timed up-and-go test will be used to look for this improvement…

The feasibilty and energy expenditure of adults with a spastic bilateral cerebral palsy playing Wii-Sports.

The aim of this study is to determine the feasibility and energy expenditure of adults with a spastic bilateral CP playing Wii-Sports.

pharmacokinetic-pharmacodynamic modeling of S(+)-ketamine in fibromyalgia

To test the hypothesis that ketamine has a modulatory effect on fibromyalgia

The effect of tetanus revaccination in patients with myasthenia gravis

The main objective of this study is to investigate the effectiveness of the humeral and cellular immune response after tetanus revaccination in patients with AChR MG, MuSK MG, or LEMS. The secondary objective is to determine if revaccination induces…

An open label, cross over pilot study to evaluate the effect of a single dose of TW001 on the oxidative stress and pharmacokinetics with and without food in patients with amyotrophic lateral sclerosis

The aim of this study is to evaluate the effect of a single oral dose of TW001 on oxidative stress and pharmacokinetics with and without food. Dosing with an oral solution is considered to have major advantages over iv dosing in a chronic condition…

Play with the IROMEC robot during rehabilitation and special education for children with severe physical disabilities - an explorative feasibility study.

This study aims to examine the feasibility and an indication of possible effects of an IROMEC robot based play therapy within rehabilitation and special education on the level of playfulness of children with severe physical disabilities.

Play with the ZORA robot during rehabilitation and special education for children with severe physical disabilities - an explorative feasibility study.

This study examines the feasibility and an indication of possible effects of ZORA robot based intervention on play in rehabilitation and special education for children with severe physical disabilities.