The information provided by BioBlast in their research indicates that they hope this treatment may prove efficacious in other SCA treatments that have similar pathology related to PolyA and PolyQ diseases.In addition, Dr. Beverly Davidson has been working on a methodology using RNAi technology to find a potential cure for over 2 decades. Her research began in the mid-1990s and progressed to work with mouse models about a decade later and most recently has moved to a study with non-human primates. The results from her most recent research "are supportive of clinical application of this gene therapy".Finally, another gene transfer technology discovered in 2011 has also been shown by Boudreau et al. to hold great promise and offers yet another avenue to a potential future cure. N-Acetyl-Leucine
N-Acetyl-Leucine is an orally administered, modified amino acid that is being developed as a novel treatment for multiple rare and common neurological disorders by IntraBio Inc (Oxford, United Kingdom).N-Acetyl-Leucine has been granted multiple orphan drug designations 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 cervical fracture, commonly called a broken neck, is a fracture of any of the seven cervical vertebrae in the neck. Examples of common causes in humans are traffic collisions and diving into shallow water. Abnormal movement of neck bones or pieces of bone can cause a spinal cord injury resulting in loss of sensation, paralysis, or usually instant death. Causes
Considerable force is needed to cause a cervical fracture. Vehicle collisions and falls are common causes. A severe, sudden twist to the neck or a severe blow to the head or neck area can cause a cervical fracture. Although high energy trauma is often associated with cervical fractures in the younger population, low energy trauma is more common in the geriatric population. In a study from Norway the most common cause was falls and the relative incidence of CS-fx increased significantly with age.Sports that involve violent physical contact carry a risk of cervical fracture, including American football, association football (especially the goalkeeper), ice hockey, rugby, and wrestling. Spearing an opponent in football or rugby, for instance, can cause a broken neck. Cervical fractures may also be seen in some non-contact sports, such as gymnastics, skiing, diving, surfing, powerlifting, equestrianism, mountain biking, and motor racing. Certain penetrating neck injuries can also cause cervical fracture which can also cause internal bleeding among other complications. Execution by hanging is intended to cause a fatal cervical fracture.