from the u.s. food & drug administration (fda) and the european medicines agency (ema) for the treatment of various genetic diseases, including spinocerebellar ataxias. n-acetyl-leucine has also been granted orphan drug designations in the us and eu for the related inherited cerebellar ataxia ataxia-telangiectasia u.s. food & drug administration (fda) and the european medicines agency (ema). published case series studies have demonstrated the effects of acute treatment with n-acetyl-leucine for the treatment of inherited cerebellar ataxias, including spinocerebellar ataxias. these studies further demonstrated that the treatment is well tolerated, with a good safety profile. a multinational clinical trial investigating n-acetyl-l-leucine for the treatment of a related inherited cerebellar ataxia, ataxia-telangiectasia, began in 2019. intrabio is also conducting parallel clinical trials with n-acetyl-l-leucine for the treatment of niemann-pick disease type c and gm2 gangliosidosis (tay-sachs and sandhoff disease). future opportunities to develop n-acetyl-leucine include lewy body dementia, amyotrophic lateral sclerosis, restless leg syndrome, multiple sclerosis, and migraine. rehabilitation physical therapists can assist patients in maintaining their level of independence through therapeutic exercise programmes. one recent research report demonstrated a gain of 2 sara points (scale for the assessment and rating of ataxia) from physical therapy. in general, physical therapy emphasises postural balance and gait training for ataxia patients. general conditioning such as range-of-motion exercises and muscle strengthening would also be included in therapeutic exercise programmes. research showed that spinocerebellar ataxia 2 (sca2) patients with a mild stage of the disease gained significant improvement in static balance and neurological indices after six months of a physical therapy exercise training program. occupational therapists may assist patients with incoordination or ataxia issues through the use of adaptive devices. such devices may include a cane, crutches, walker, or wheelchair for those with impaired gait. other devices are available to assist with writing, feeding, and self care if hand and arm coordination are impaired. a randomised clinical trial revealed that an intensive rehabilitation program with physical and occupational therapies for patients with degenerative cerebellar diseases can significantly improve functional gains in ataxia, gait, and activities of daily living. some level of improvement was shown to be maintained 24 weeks post-treatment. speech language pathologists may use both behavioral intervention strategies as well as augmentative and alternative communication devices to help patients with impaired speech.