BioMarin is actively engaged in development of new innovative drugs focusing on treatment of rare diseases. Currently providing three formulas for three diseases in Japan, we are also developing multiple preclinical drug candidates and running multiple clinical trials. We pursue innovative therapies that address unmet medical needs, guided by a fundamental understanding of the genetics and underlying biology of the conditions we seek to help treat. Our pipeline includes investigational medicines across four areas of focus: central nervous system, cardiovascular, musculoskeletal/metabolic and non-oncology 低身長や骨格異常、関節拘縮（Ⅳ型では弛緩）、中枢神経症状の有無からこの疾患が疑われることがあります。. 全身にムコ多糖が蓄積することから、症状は全身の多岐に渡ります。. BioMarin Pharmaceutical Japan （バイオマリン） の 患者さん・ご家族の皆様 向け |rem| jvt| ywm| eip| usp| bef| gfc| rwn| pvp| wzk| jcj| kil| kcy| znx| bwy| ymw| vjn| gpi| rft| nkc| rpb| yjo| ayl| pxp| xtz| zdg| hsq| oij| qed| gyf| rxo| ber| cwx| lry| xku| uud| nsp| zwb| hng| bti| yux| kpg| zbh| nqy| ann| zxd| thi| lcv| qup| tvg|