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 »
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 »
Novartis Gene Therapies has provided an update to the SMA Community. Check out the latest here!
SMA Community Update from Novartis Gene Therapies Read More »
While sibling relationships aren’t always easy, Alexandra ‘Lexi’ Lakhman and her brother Joe have always been close. Despite Lexi being six years older than Joe, she grew up viewing Joe as a role model, particularly for his positive outlook on life. Joe was diagnosed with spinal muscular atrophy (SMA) Type 2 in 2002 when there
Community Spotlight: Alexandra and Joe Lakhman Read More »
Every August, the spinal muscular atrophy (SMA) community unites, raises awareness, and shows our pride for SMA Awareness Month. This year, Shaakira Thomas and her family chose to go above and beyond by organizing landmark lightings all over the country, all in support Cure SMA’s newborn screening initiatives. Shaakira and her husband, Parris Sr., are parents to Aaliyah,
Community Spotlight: Shaakira Thomas and Family Read More »
On the 25th anniversary of the SMA Researcher Meeting, we would like you to learn more about Dr. Burghes and his commitment to the Cure SMA community. Arthur Burghes, Ph.D., is a Professor of Biological Chemistry and Pharmacology at The Ohio State University and an internationally recognized research scientist who has dedicated more than 30 years
Community Spotlight: Dr. Arthur Burghes Read More »
Biogen today announced new results from the NURTURE study, adding data to the longest study of spinal muscular atrophy (SMA) in pre-symptomatic infants (n=25). These data reported, after up to 45.1 months of analysis, continue to demonstrate efficacy and safety in patients treated pre-symptomatically with SPINRAZA in comparison to the natural history of this disease.
Biogen Announces New NURTURE Data at the 2019 Annual SMA Conference Read More »
We’ve recently released an update to the SMA drug pipeline. This latest version includes: 23 active programs, including two approved therapies. 15 pharmaceutical partners. 6 programs in clinical trials. An ever-increasing breadth of potential treatment approaches to SMA. Effective Treatments for All Ages, Stages and Types of SMA The updated version of this pipeline reflects
Updated SMA Drug Pipeline Now Available Read More »
