The purpose of this study is to obtain information pertaining to the occurrence of antibodies
to investigational SPK-3006 and GAA, GAA activity and GAA antigen levels in the usual care
setting of Late-Onset Pompe Disease (LOPD) participants on an enzyme replacement regimen.
Additionally, a careful evaluation of laboratory and functional testing in patients with LOPD
may provide information to better understand the disease features and better drive the design
of a future interventional investigational gene therapy trial. An understanding of the
underlying status of liver and muscle health in individuals with LOPD may also inform best
surveillance during the conduct of gene therapy trials.

<p>Brain activity likely to be involved in control of brain-computer interfaces (BCI) will be recorded by electroencephalography (EEG), a non-invasive technique. These recordings will be used to control a computer-based augmentative and alternative communication (AAC) device.

This is a Phase 1, double-blind, placebo-controlled, single ascending dose cohort study to
evaluate the safety, tolerability, and plasma concentrations of WVE-210201 in ambulatory and
non-ambulatory male pediatric patients with DMD amenable to exon 51 skipping intervention.

The TopCSPN trial is a double blinded randomized placebo controlled study of oral topiramate
as a potential disease altering therapy for cryptogenic sensory peripheral neuropathy (CSPN).
Patients with CSPN who also have metabolic syndrome (defined by the ATPIII criteria) who do
not have an alternative cause for neuropathy will be potentially eligible. The co primary
outcome measures are change in the Norfolk Quality of Life - Diabetic Neuropathy (NQOL-DN)
Scale and intraepidermal nerve fiber density (IEFND) at the distal thigh. The treatment phase
will last 24 months.

By doing this study, researchers hope to learn the effects of aerobic exercise on heart,
lung, and bone health in people who cannot walk after a stroke.

The purpose of this study is to learn the effects of two mild body exercises on quality of
life, non-motor symptoms, anxiety, depression, fatigue, sleep quality, cognition, and
executive function on people with Parkinson's Disease (PD).

This study seeks to evaluate the efficacy and safety of ABBV-8E12 in subjects with Early
Alzheimer's Disease.

The primary cause of facioscapulohumeral muscular dystrophy (FSHD), a common adult-onset
dystrophy, was recently discovered identifying targets for therapy. As multiple drug
companies pursue treatments for FSHD, there is an urgent need to define the clinical trial
strategies which will hasten drug development, including creating disease-relevant outcome
measures and optimizing inclusion criteria. This proposal will develop two new outcome
measures and optimize eligibility criteria by testing 160 patients in 7 sites over a period
of 18 months.

This study is a 4-week, multicenter, randomized, double-blind, placebo-controlled, ascending
dose, 4-period crossover study designed to evaluate the safety, tolerability, efficacy, and
PK of JZP-110 (75, 150, and 300 mg) in the treatment of excessive sleepiness in adult
subjects with idiopathic PD.

A study of a drug to be used in addition with another drug to treat adults with Uncontrolled
Partial-onset Seizures

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