Macedonian Gaucher Activist For Rare Disease Patients & Getting Ready For The Cold
We discuss a Macedonian Gaucher activist, whose publicizing the plight of rare disease patients. Although fall and winter are her least favorite seasons, DeAnn finds being prepared can help the transi...
30 Syys 20198min
European Spinraza Label Updated to Reflect Long-Term Benefits & Trending Topics
We discuss how the European Spinraza label was updated to reflect long-term benefits in broad ranges of SMA patients and Community Development Manager Kevin Schaefer talks about some of the trending t...
27 Syys 20197min
High Chance Of Scoliosis Surgery in Types 1C and 2 & Superheroes With Disabilities
SMA News Today’s Director of Multichannel Content, Michael Morale, discusses a Dutch study that states there is a high probability of scoliosis surgery found for SMA types 1C, and 2. Community Develop...
25 Syys 20198min
Spinraza Gives Babies With Type 1 SMA Hope For a Healthy Life in North Macedonia
We discuss how Spinraza gives babies with type 1 SMA in North Macedonia hope for a healthy life. Also, SMA News Today’s forums moderator, DeAnn Runge, shares how service dogs were introduced into her ...
23 Syys 20196min
Phase 2 Trial Shows Presymptomatic Spinraza Treatment to SMA Children Reaching Milestones
Community Development Manager Kevin Schaefer talks about some of the trending topics in the SMA News Today forums. We discuss how a phase 2 trial shows presymptomatic Spinraza treatment leads to SMA c...
20 Syys 20196min
The World’s First Alport Stamp is a Macedonian Mom’s Latest Win for Rare Disease Patients
Community Development Manager Kevin Schaefer talks about some of the trending topics in the SMA News Today forums. SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how the ...
18 Syys 20199min
#42 - Interview With Kyle Harris
In episode 42, host Kevin Schaefer talks with Kyle Harris from San Diego, California. Kyle talks about growing up with SMA Type 3, college, and accessibility in the modern world. Are you interested i...
17 Syys 201936min
RNA Therapy Delivered by Viral Vector May Hold Potential to Treat SMA
We discuss how an RNA therapy that’s delivered by a viral vector may hold potential to treat SMA. Also, SMA News Today’s forums moderator, DeAnn Runge discusses how airlines do not allow wheelchair us...
16 Syys 20198min
