Capillary Malformation-Arteriovenous Malformation (CM-AVM)

The purpose of this study is to identify patients with a diagnosis of Amyotrophic Lateral Disease (ALS) or ALS variants who have laboratory markers of inflammation or autoimmunity. Presently, there is no effective therapy for treatment of ALS or ALS variants. Recent evidence suggests that some patients with ALS who have laboratory markers of inflammation may respond to therapies that modulate immune system function. The aim of this study is to determine what percentage of patients with ALS and ALS variants have positive laboratory markers of inflammation or autoimmunity. This study will also determine what percentage of patients with ALS or ALS variants chose to receive immunotherapy. Furthermore, the study will determine what percentage of patients with ALS or ALS variants respond to immunotherapy and which specific forms of immunotherapy are effective.  Finally, the study will determine the duration that positive markers of inflammation remain elevated during the year of observation and if immunotherapy affects the results of laboratory markers of inflammation.

The purpose of this study is to evaluate the effect of retigabine dosages on motor neuron activity in people with Amyotrophic Lateral Sclerosis (ALS).

This first-in-human, double-blind, placebo-controlled Phase I study will be conducted in participants with amyotrophic lateral sclerosis (ALS) to explore safety, tolerability, and pharmacokinetic (PK) properties of GDC-0134. It will include two components: a Single-Ascending-Dose (SAD) stage and a Multiple-Ascending-Dose (MAD) stage.

The purpose of this study is to evaluate the safety and effectiveness of NP001 for treating patients who have amyotrophic lateral sclerosis (ALS) and evidence of systemic inflammation.

The goals of this study are to identify biomarkers that allow improved staging or prognosis of the disease, and through the discovery of previously unrecognized immune abnormalities in ALS we aim to eventually identify immune therapies that may provide benefit in ALS.

This study is intended to obtain clinical information and establish a repository of DNA, RNA, peripheral blood monocyte, lymphocyte and skin tissue samples from people with ALS and related neurodegenerative motor neuron diseases, people with a family history of these conditions, and healthy people with no family history of these disorders. The samples will be used in future research to learn about how these disorders affect people, what causes these conditions, and how the investigators can tell when someone has this kind of disease. Future research may also include the generation of stem cells from stored blood cell and skin cell samples. Participants will not be paid for taking part in this study.

The purpose of this study is to collect and study blood samples that can be used in current and future research studies to identify genetic risk factors in ALS and identify potential biomarkers in blood collected in ALS patients. Biomarkers are non-genetic elements in your blood that may help us diagnose and monitor ALS more easily. There are no readily available sources of longitudinal DNA, RNA, monocytes, serum or plasma from people with ALS, ALS-FTD or similar neurodegenerative disorders or their family members for use in the identification of potential changes in gene structure over time or biomarkers in ALS. Future research will examine genes associated with the risk of ALS and potential biomarkers in blood collected over time to see if they change over time and can be used to diagnose and monitor people with ALS. This information is important for development of new medications for the treatment of ALS.

The purpose of this study is to assess the safety and effectiveness of NP001 for the treatment of patients who have amyotrophic lateral sclerosis (ALS) and evidence of systemic inflammation.