Editor’s note: This is an automatically generated transcript. Please notify [email protected] if there are concerns regarding accuracy of the transcription. So for spinal muscular atrophy, we have ...

The therapy’s safety profile was consistent across both trials, with fewer than 2% of participants discontinuing treatment due to adverse events. Credit: Prostock ...

Mary is the bedrock of her family. “She’s the one we all go to,” says her son Eric. “She’s selfless, extremely sweet, very personable—but she can also be very feisty.” So when she returned from an eye ...

Vitamin supplements known to potentially slow the progression of intermediate age-related macular degeneration do not seem to have a similar effect on geographic atrophy, an advanced form of the dry ...

While pivotal research on therapies for neovascular age-related macular degeneration (nAMD) and geographic atrophy (GA) focused on one condition or the other, retina specialists commonly encounter ...

Injections for spinal muscular atrophy (SMA) refer to two treatments that the Food and Drug Administration (FDA) has approved for SMA: nusinersen (Spinraza) and onasemnogene abeparovec-xioi (Zolgensma ...

Oral risdiplam (Evrysdi, Genentech) started in the first 6 weeks of life let most infants with presymptomatic spinal muscular atrophy (SMA) reach motor milestones typical of healthy babies, results of ...

Risdiplam, an oral pre–messenger RNA splicing modifier, is an efficacious treatment for persons with symptomatic spinal muscular atrophy (SMA). The safety and efficacy of risdiplam in presymptomatic ...

Trypanosoma evansi is the etiological agent of the disease known as “mal das cadeiras” or “surra” that affects domestic and wild animals (Silva et al. 2002), with reports of several clinical and ...

Spinal muscular atrophy with respiratory distress (SMARD) is a rare genetic condition that typically affects infants and children. It causes muscle weakness and breathing problems. Spinal muscular ...

Panelists discuss how spinal muscular atrophy is an autosomal recessive genetic disease affecting motor neurons with 3 currently approved disease-modifying therapies that restore SMN protein ...

The causes of male infertility and reproductive aging remain largely elusive. This study identifies a necroptotic pathway, initiated by stress granule formation and mediated by ZBP1 and RIPK3, as a ...