ArmaGen is focused on developing revolutionary therapies for severe neurological disorders. The company is advancing innovative therapies for the treatment of currently unaddressed neurological complications of lysosomal storage disorders (LSDs), as well as neurodegenerative diseases such as Alzheimer’s and Parkinson’s. We conduct clinical trials independently and in collaboration with industry partners to assess the safety and efficacy of investigational medicines, which if proven, will allow us to obtain the necessary approvals from regulatory authorities to provide patients with broad access to these medicines.
In general, ArmaGen believes that participating in clinical trials is the best way for patients to access medicines prior to approval. In some extreme circumstances when this is not possible, patients with life-threatening diseases or conditions may seek special access to investigational medicines outside of a clinical trial setting. These situations are typically referred to as compassionate use, but can also be known as expanded access, early access, pre-approval access and emergency use.