Updates
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I Don’t Need to Win to Be a Winner: Volunteering Is Its Own Reward
My parents and I recently attended a National Philanthropy Day breakfast with a group of staff from Turnstone, our local center for people with disabilities and ... Read more -
The Unexpected Feeling a Bookstore Gave Me
I was recently strolling through the best-sellers section of a bookstore and couldn’t remember the last time I had set foot in one. I ... Read more -
HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases
Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access ... Read more -
Tiny Shoes Unleash a Flood of Emotions
Our third baby, Jeffrey, shook things up from the moment the nurse at the doctor’s office exclaimed, “You’re pregnant! You’re very pregnant!” She seemed ... Read more
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Spinraza Stabilizes or Improves Motor Function in Older SMA Patients, US Real-world Study Suggests
Spinraza (nusinersen) safely stabilizes or improves motor function in older patients with spinal muscular atrophy ... -
Ella Sings After Disappointment Dissipates
The holidays are upon us. In our house, Christmas starts the day after Thanksgiving. ... -
Dealing with Inevitable Chest Infections During Cold and Flu Season
At the beginning of November, I got sick. It was inevitable, though I had ... -
My 10-year Challenge: Reflections on a Decade
If you’ve been on social media lately, you’ve probably seen a flood of posts ... -
Multidisciplinary Approach Improves Spinraza Dosing Process in SMA Patients, US Study Shows
Adopting a multidisciplinary approach can optimize pretreatment evaluations and improve the dosing process for ... -
Ella Faces Disappointment with a Choir Solo Part
Life can be full of disappointments. They are opportunities for children to learn how ...
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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 to ... Read more