For the treatment of Spinal and Bulbar Muscular Atrophy (SBMA), preliminary pilot studies reveal small molecule clenbuterol is effective at improving motor function in SBMA. Research will continue with Phase II clinical trial NCT06169046 soon, as it is not yet recruiting, to research the safety and efficacy of clenbuterol as a treatment for SBMA. Another small molecule, AJ201 has been granted FDA orphan drug designation for the treatment of SBMA, HD, and SCA. A Phase I safety study in healthy volunteers was successfully completed in 2021. A Phase I/Phase II study (NCT05517603) is currently active evaluating safety, tolerability, pharmacokinetics, and pharmacodynamics of AJ201 for the treatment of SBMA. Lastly Dentatorubral-pallidoluysian atrophy (DRPLA) only has one registered clinical trial. This trial (NCT05489393) will establish a global patient registry consisting of a database with patient reported data on individuals affected with DRPLA from around the world.