More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the rare disease community. Hosted by the EveryLife Foundation’s Rare Disease Legislative Advocates (RDLA) program, the event brings together community members from across the U.S. to…
Photo courtesy of Rachel Stewart Day 2 of 31 This is Rachel Stewart’s story: Growing up, I didn’t have any role models with my disability, let alone know someone with SMA who was married, or in a queer relationship. And I certainly didn’t see any representation on TV or…
Photo courtesy of Alvaro Cheherlian Day 1 of 31 This is Alvaro Cheherlian’s (@_hotwheelz) story: Being an entrepreneur is hard. Being an entrepreneur with SMA adds challenges that make it even harder, but I didn’t let that stop me. My name is Alvaro Cheherlian, I have type…
SMA News Today’s 31 Days of SMA campaign will publish one story per day from someone who has been affected by the disease for SMA Awareness Month in August. Stay tuned for the first story in the series starting on Aug. 1, and follow us on Facebook and Instagram for…
The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing, in a joint statement with 26 other U.S. patient organizations. The interim final rule will implement patient protections required by the No Surprises Act. Surprise…
AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies are those used to treat rare and chronic conditions in the U.S., and are often extremely costly. For…
Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work to make a difference for the global rare disease community. The deadline for nominations is Sept. 10…
SMA News Today is conducting a U.S.-based survey to better understand the characteristics, needs, and treatment experiences of the spinal muscular atrophy (SMA) community. Adults — from patients to clinicians — ages 18 and older are invited to participate. The survey is intended to take no more than 20-25…
A recent survey showed that spinal muscular atrophy (SMA) patients and their families are generally supportive of genetic screening program for early detection of SMA. However, they still have a number of fears about the real outcome of the tests. These findings stressed the need of improved relations between those…
On the eve of the weekend Christmas holiday, the U.S. Food and Drug Administration (FDA) approved Biogen’s therapy Spinraza (nusinersen) for the treatment of spinal muscular atrophy in both children and adults. The landmark approval marks the first time the federal agency has approved a therapy that directly treats the orphan disease, which is…
