| 摘要 |
<p>The disclosure relates to methods and compositions (e.g., compounds and pharmaceutical compositions thereof) useful for increasing expression of SMN in a cell (e.g., in vitro or in vivo). As a deficiency in SMN can result in the development of an SMA condition in a subject, the methods and compositions described herein can also be used to, e.g., treat, or prevent, an SMA condition in a subject.</p> |
| 申请人 |
FAMILIES OF SPINAL MUSCULAR ATROPHY;RUTGERS, THE STATE UNIVERSITY OF NEW JERSEY;THE OHIO STATE UNIVERSITY;JARECKI, JILL;SINGH, JASBIR;GURNEY, MARK;POLLOK, BRIAN;MICHAUD, GREGORY;LIU, SHIN-WU;KILEDJIAN, MEGERDITCH;BUTCHBACH, MATTHEW;BURGHES, ARTHUR |
发明人 |
JARECKI, JILL;SINGH, JASBIR;GURNEY, MARK;POLLOK, BRIAN;MICHAUD, GREGORY;LIU, SHIN-WU;KILEDJIAN, MEGERDITCH;BUTCHBACH, MATTHEW;BURGHES, ARTHUR |
