Updates
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How a Stranger’s Words Pulled Me Out of a Rut
I always struggle with mental health at this time of year. Winter has that effect on me. Each year, I tell myself things will ... Read more -
ArcherDX Enters Newborn Screening Market with Baby Genes Acquisition
ArcherDX is expanding its product portfolio with newborn and screening tests to identify carriers of genetic variants linked to different disorders, including cystic fibrosis, ... Read more -
Scotland Opens Spinraza to All SMA Patients Using Its Public Health System
Scotland has joined the growing roster of countries making the spinal muscular atrophy (SMA) therapy Spinraza available to patients at low or no ... Read more -
Ella’s Smooth Clinic Appointment
An early morning rain lent itself to the Chicago traffic. The highway was backed up for miles and we had an hour to get ... Read more -
New Compound Improves Clinical Outcomes in Mouse Models of SMA, Study Shows
A new molecular compound, called LDN-2014, increases and stabilizes the levels of the survival motor neuron (SMN) protein, improving clinical outcomes in mouse models of mild ... Read more
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Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide
Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities ... -
Finding Joy Amid Career Changes
It’s been a hard week. Half because I’m an emotional kiddo who takes everything ... -
Sense of Well-Being Tied to Ability to Take Part in Life and Engage with Others, SMA Patients Say
For people with spinal muscular atrophy (SMA), psychological well-being is not driven by socioeconomic factors ... -
On Hiring New Caregivers
Recently, one of our columnists, Kevin Schaefer, took to the SMA forums to discuss ... -
New SMArtCARE Online SMA Registry Will Gather Patient Data
Researchers have developed a platform called SMArtCARE that allows prospective monitoring of all spinal ...
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Researchers Discover Promising New Gene Therapy Approach for Spinal Muscular Atrophy
News
A new genetic approach to restore the expression of the gene that causes spinal muscular atrophy (SMA) in patients’ cells holds promise to effectively and permanently treat the disease, researchers suggest. The study, “Seamless genetic conversion of SMN2 ... Read more