Cure SMA will host a webinar on spinal muscular atrophy (SMA) today at 1 p.m. EST. The event focuses on access to Spinraza — the only U.S. government-approved therapy to treat the disease, CureSMA said in a press release.
Topics to be covered include the current status of dosing in individual U.S. states and information on where the drug is being administered — including a list of all sites — as well as commercial and government insurance policies regarding Spinraza, and other community resources and support programs.
Spinraza, developed by Biogen, was shown in laboratory and preclinical studies to boost levels of SMN (survival motor neuron) protein by increasing expression of the survival motor neuron 2 (SMN2) gene. In clinical studies, Spinraza improved motor function in SMA patients compared to untreated individuals. In those studies, Spinraza also helped patients live longer, achieve milestones such as sitting, standing and walking — and maintain those milestones as patients aged.
Attendees must register here. Cure SMA encourages participants to pre-submit questions upon registration so that it can answer as many as possible, though attendees can also ask questions during the webinar as there will be an expanded question-and-answer period.
“We look forward to hosting an open, candid and informal discussion of where our community stands on issues surrounding access to this first-ever approved therapy, and what our next steps will be,” the organization said.
August was SMA awareness month, which the organization marked in several ways, including advocacy visits to a dozen Senate and House congressional offices to advocate for issues that affect people with the disease. In particular, it urged more federal funding for newborn screening. Even such programs are generally run at the state level, the federal government can also play a role in influencing decisions.
Families and Cure SMA leaders across the nation also asked their local municipalities and states to issue proclamations of SMA awareness month. Candlelight vigils were also held to remember those who have died from SMA and honor those living with the disease. In addition, they also held fundraisers and coordinated baseball games to raise public awareness of SMA.