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…
