Week in review: Mouse debut, disabled docs, innate confusion

A weekly roundup of ophthalmic news from around the web.

Luxturna carries a hefty price tag, but it’s not actually the most expensive drug in the United States. GoodRx ranked drugs by their annual cost for a typical course of therapy, based on each drug’s list price. At $850,000, Luxturna placed second. The nation's most expensive drug is ... drumroll please ... Novartis’ Zolgensma, a $2,125,000 per year treatment for spinal muscular atrophy. GoodRx

Mice carrying the keratoconus mutation PPIP5K2 made their debut in the laboratory of Yutao Liu, MD, PhD, a geneticist working on a $2.1 million grant from the National Eye Institute to study the condition’s causes. Like humans with keratoconus, the model mice develop a rough corneal curvature and thickened epithelial layer. The rodent’s compressed lifespan, however, gives scientists time to monitor changes and uncover pivotal points to intervene in the disease process. Augusta University JAGWIRE

An ophthalmology researcher with a prolific career—and extremely limited vision due to myopic macular degeneration—says medicine has a lot to learn from doctors with disabilities. NPR sat down with Bonnielin Swenor, PhD, MPH, an assistant professor of ophthalmology at the Wilmer Eye Institute, to talk about the importance of representation, the challenges she faces as a clinical researcher with limited vision and the surprising upsides to having a disability in the clinic. NPR

An anti-angiogenic, anti-scarring agent for wet AMD sailed through phase 1/2a safety tests, RIBOMIC reports. A single dose of the company's experimental drug RBM-007 improved vision or central retinal thickness in 7 of 9 patients with a history of poor anti-VEGF response; no safety concerns emerged. The drug targets fibroblast growth factor 2 and may offer an added benefit or alternative to anti-VEGF regimens for patients with wet AMD. RIBOMIC

The complement system often gets a bad rap when it comes to retinal degeneration, but new findings suggest there’s more to the story. Although complement activation happens at the exact time and place of photoreceptor degeneration, it plays a helpful—not harmful—role by clearing away dead cells and promoting physiological balance, a National Eye Institute-led team found. “Our findings suggest that [complement inhibitors] may be appropriate for some disease scenarios but may induce complex responses in other disease scenarios by inhibiting helpful and homeostatic functions of inflammation,” said lead investigator Wai T. Wong, MD, PhD. NEI, Journal of Experimental Medicine