As therapies are supported by proven effectiveness that can be repeated in multiple studies, they are useful, but the clinician may also consider that the best treatments for patients can only be evaluated on a case-by-case basis. It is the interplay of these two facets of neurology and medicine that may bring about significant progress in this field. See also
Basal ganglia disease

References


== External links ==
Researchers measured the participants annualized growth velocity, or rate of height growth, at the end of the year. Participants who received vosoritide grew an average 1.57 centimeters taller compared to those who received a placebo. The U.S. Food and Drug Administration (FDA) granted the approval of Voxzogo to BioMarin. Society and culture
Controversy
Some people with achondroplasia, as well as parents of children with this condition, have reacted to vosoritides study results by saying that dwarfism is not a disease and consequently does not need treatment. Research
Vosoritide has resulted in increased growth in a clinical trial with 26 children. The ten children receiving the highest dose grew 6.1 centimetres (2.4 in) in six months, compared to 4.0 centimetres (1.6 in) in the six months before the treatment (p=0.01). The body proportions, more specifically the ratio of leg length to upper body length – which is lower in achondroplasia patients than in the average population – was not improved by vosoritide, but not worsened either.As of September 2015, it is not known whether the effect of the drug will last long enough to result in normal body heights, or whether it will reduce the occurrence of achondroplasia associated problems such as ear infections, sleep apnea or hydrocephalus. This, together with the safety of higher doses, is to be determined in further studies. References
External links
"Vosoritide". Drug Information Portal. U.S. National Library of Medicine. Clinical trial number NCT03197766 for "A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia" at ClinicalTrials.gov