You know that poem by Anne Boyer I love about holes? You fall into one, only to dig yourself out over weeks or months or years, only to fall into yet another hole, this one both different and familiar. Life, then, becomes a series of holes, some small…

After a “constructive and collaborative in-person” meeting with the U.S. Food and Drug Administration (FDA), Scholar Rock is now planning to once again seek approval of its muscle-strengthening agent apitegromab as a treatment for spinal muscular atrophy (SMA). That resubmission to the FDA is expected in…

The experimental therapy BIO101 may help improve muscle strength in people with spinal muscular atrophy (SMA), according to new research done in laboratory models. “We demonstrated that a daily oral administration of BIO101 can specifically improve muscular function in severe (infanthood) and mild (adulthood) … SMA mouse models,” the…

According to my mother, I made sure she was being “kessul” (careful) during her pregnancy with my brother. I wasn’t thrilled, however, about sharing the spotlight with baby Paul. Thinking outside the box, even then, I asked sweetly to push Paul around in the buggy. My folks, weary from parenting…

Treatment with Spinraza (nusinersen) may help stabilize lung function in people with spinal muscular atrophy (SMA) types 2 and 3, a study suggested. “Treatment of SMA types 2 and 3 with nusinersen may stabilize respiratory muscle strength and delay [noninvasive ventilation] dependence, despite progressive restrictive lung disease,” the…