HHS Secretary's Efforts To Cure Rare Diseases & Taking Advantage of New Opportunities
We discuss the HHS Secretary, Alex Azar, touts the White House efforts to cure rare diseases & Forums Director Kevin Schaefer talks about taking advantage of new opportunities when you have SMA. Are ...
24 Jan 20208min
Being Rare and Resilient & No Topic Is Off Limits on The SMA News Today's Forums
Go to our forums: http://bit.ly/forumsflashbriefings Forums Director Kevin Schaefer recounts the story of his adventures in Philadelphia, and talks about what it means to be rare and resilient. Also,...
22 Jan 20207min
#50 - Roundtable Discussion - January 2020
In episode 50, hosts Kevin Schaefer and Michael Morale discuss updates on Roche and Genentech’s experimental therapy risdiplam. Now that this drug is under FDA-approval, we answer questions regarding ...
21 Jan 202035min
Spinraza Stabilizes or Improves Motor Function in Older SMA Patients & The Reality of Aging
We discuss a US real-world study, that claims that Spinraza stabilizes or improves motor function in older SMA patients. Also, when you have a disease that’s considered the number one genetic killer o...
20 Jan 20208min
Multidisciplinary Approach Improves Spinraza Dosing Process in SMA Patients & Parenting and SMA
We discuss a study that states how a multidisciplinary approach improves the Spinraza dosing process in SMA patients. Also, Forums Director Kevin Schaefer reads the latest piece by parent columnist Mi...
17 Jan 20209min
Importance of Media Representation for People With Disabilities & Conversations in the SMA Forums
Forums Director Kevin Schaefer talks about the importance of media representation for people with disabilities, and calls for more of it in 2020. Also, great conversations are going on in the forums. ...
15 Jan 20206min
Targeted Genetic Screening Helps Diagnosing Patients With SMA-Like Symptoms & Pranking With SMA
We discuss how targeted genetic screening may be useful in diagnosing patients with SMA-like symptoms. Also, a harmless prank can be fun, but it’s not always easy when you have SMA. With the help of h...
13 Jan 20207min
Early Trial Data Shows SRK-015 Increases Myostatin Growth Factor Levels in SMA Patients
We discuss early trial data that shows SRK-015 increases myostatin growth factor levels up to 100-fold in SMA patients & Forums Director Kevin Schaefer reads an excerpt from Alyssa Silva’s latest colu...
10 Jan 20207min
