Play is important for kids. It allows children to develop cognitive, social, emotional, and physical skills while engaging creatively. But it’s not just how kids play that’s important, what they play with also matters. Which is why Michelle Tynski, mom to nine-year-old Trace, Benjamin who passed away in 2016, and 3-year-old Zach, who has Type […]
Community Spotlight: Michelle Tynski and Family Read More »
Genentech, announced earlier this month new two-year data from the JEWELFISH study evaluating Evrysdi® (risdiplam) in people with Type 1, 2 or 3 SMA aged 6 months to 60 years at time of enrollment. Patients had been previously treated with other approved or investigational SMA-targeting therapies, including Spinraza® or Zolgensma®. Data showed Evrysdi improved or maintained motor function
Fifty years ago today, the Supplemental Security Income (SSI) program was created to “provide supplemental security income to individuals who have attained age 65 or are blind or disabled” for their basic living needs, such as housing, clothing, medical costs, and food. The program and its monthly cash benefit have been a lifeline for many
SMA Community Seeks SSI Improvements on Program’s 50th Anniversary Read More »
Scholar Rock yesterday announced new quality-of-life (QoL) data from its Phase 2 TOPAZ trial extension period evaluating patient outcomes after 24 months of treatment which indicate stabilization or continued improvement with apitegromab for nonambulatory patients with Types 2 and 3 spinal muscular atrophy (SMA) receiving an SMN-targeted therapy. The TOPAZ trial assessed activities of daily
The Igbo and Yoruba proverb, “It takes a village to raise a child” has been in use for centuries and remains in use across languages and cultures due to its continued relevance. For Amber-Joi Watkins, her husband, Tommy Domalski, and their daughter, Céline, that village includes family and friends, as well as Céline’s pediatrician, her
Community Spotlight: Amber-Joi Watkins and Family Read More »
On Thursday, August 4, 2022, six members of the SMA community had the opportunity to talk directly with the U.S. Food and Drug Administration (FDA) about their lived experiences with SMA and their unmet medical needs. This Patient-Led Listening Session was a private teleconference attended by patient advocates, FDA representatives from the Center for Drug
Cure SMA Holds Patient-Led Listening Session with FDA Read More »
Nothing brings people together more than sharing a meal, but what if you could make that meal even more meaningful? We’re encouraging all members of our SMA community to consider hosting a dining fundraiser, where a portion of purchases are designated to support Cure SMA. This is also a great way to supplement your efforts
A Deliciously Easy Way to Fundraise for Cure SMA Read More »
On Thursday, August 4, 2022, six members of the SMA community had the opportunity to talk directly with the U.S. Food and Drug Administration (FDA) about their lived experiences with SMA and their unmet medical needs. This Patient-Led Listening Session was a private teleconference attended by patient advocates, FDA representatives from the Center for Drug
Cure SMA Holds Patient-Led Listening Session with FDA Read More »
Nothing brings people together more than sharing a meal, but what if you could make that meal even more meaningful? We’re encouraging all members of our SMA community to consider hosting a dining fundraiser, where a portion of purchases are designated to support Cure SMA. This is also a great way to supplement your efforts
A Deliciously Easy Way to Fundraise for Cure SMA Read More »
Cure SMA is pleased to announce that South Carolina is now screening babies born in the state for spinal muscular atrophy (SMA). South Carolina Department of Health and Environmental Control (DHEC) officials shared the exciting news with Cure SMA in a written statement earlier today: Good news! The new multiplex PCR based method for screening
Special NBS Awareness Month Announcement: South Carolina Screens for SMA! Read More »
