The purpose of this study is to learn about the effects of an investigational medicine, PGN-EDODM1, to see how safe and tolerable multiple administrations of PGN-EDODM1 are for people with myotonic dystrophy type 1 (DM1) compared to placebo.
A phase III, randomized, multi-center, investigational, open label clinical trial that will examine whether treatment with endovascular thrombectomy is superior to standard medical therapy alone in patients who suffer a Distal Medium Vessel Occlusion Ischemic Stroke within 12 hours from time last seen well
This EAP will provide access to pridopidine for up to 200 patients with ALS who are ineligible for clinical trials. Pridopidine will be given at a dose of 45 mg twice daily p.o. (or via feeding tube).
Each patient will be followed for 2 years with regularly scheduled visits. The screening and baseline visits will be performed in person; subsequent visits may occur in person or remotely. Recommended in-person visits will occur at Weeks 12, 52, 78, and end of treatment (Week 104 or early termination). If the patient is unable to complete the visits in person, these visits may also be completed remotely.
RAPA-501-ALS is an Intermediate-Size Expanded Access Trial of RAPA-501 autologous hybrid TREG/Th2 cells in patients living with amyotrophic lateral sclerosis (pwALS).
An Intermediate Expanded Access Protocol (EAP) with CNM-Au8 for Amyotrophic Lateral Sclerosis for NIH Grant RFA-NS-23-012