NORD Honors Industry In Patient Advocates At Rare Impact Awards Gala & Overprotective Parents
We discuss how NORD honors industry in patient advocates at rare impact awards gala and SMA News Today forums moderator DeAnn Runge shares her perspective on overprotective parents. A college friend is coming to visit and instead of allowing her friend, who also has a disability, to do this on her own her mother is tagging along. DeAnn hopes in leading by example her friends Mom will find a better balance between support and independence with her daughter.
2 Syys 20198min
Anti-Epileptic Keppra Can Be A Candidate For SMA Treatment & Batcave For Personal Space
We discuss a study that suggests that suggests Anti-epileptic Keppra could be a candidate for treatment of SMA and Community Development Manager Kevin Schaefer reads his latest column about acquiring personal space, via his Batcave.
30 Elo 20198min
Zolgensma’s Arrival Adds Urgency to SMA Newborn Screening Efforts & Natalie Russo And Her Brother
SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how Zolgensma’s arrival adds urgency to SMA newborn screening efforts in the US and Community Development Manager Kevin Schaefer reads one of the final 31 Days of SMA stories. In this story, Natalie Russo talks about her and her brother having SMA.
28 Elo 20199min
Eurordis' Integrated Care Initiative For Rare Disease Patients & Cooperate With Physical Therapists
We discuss how Eurordis unveils an integrated care initiative for rare disease patients. Also, SMA News Today’s Director of Multichannel Content, Michael Morale, discusses working with physical therapists, and making them more comfortable while working out with patients with SMA. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it is being investigated for the treatment of genetic diseases. Visit www.exploregenetherapy.com
26 Elo 20199min
RNA Molecule miR-23a May Have Therapeutic Role in SMA & From College Writing Course To A Career
We discuss a study that suggests that the small RNA molecule miR-23a, may have a therapeutic role in SMA. Also, Community Development Manager Kevin Schaefer reads from his latest column. In this post, he reflects on how a college writing course prepared him for his career. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it is being investigated for the treatment of genetic diseases. Visit www.exploregenetherapy.com
23 Elo 20198min
Zolgensma’s Price And Its 'Cure One, Cure Many' Potential & Concerts Accessibility
SMA News Today’s Director of Multichannel Content, Michael Morale, discusses an interview where researchers talk about Zolgensma’s price, and its cure one, cure many potential. Also, Community Development Manager Kevin Schaefer reads from the latest 31 Days of SMA story. This post focuses on concert accessibility. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it is being investigated for the treatment of genetic diseases. Visit www.exploregenetherapy.com
21 Elo 20199min
#40 - Interview With Kellie Cusack
In episode 40 of the SMA News Today Podcast, host Kevin Schaefer interviews Kellie Cusack. Kellie lives in New Jersey with her family, and is currently studying journalism at a community college. She talks about growing up with SMA Type II, blogging, and her involvement with the SMA community. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it is being investigated for the treatment of genetic diseases. Visit www.exploregenetherapy.com
20 Elo 201928min
High Levels of Alpha-COP Proteins Seems To Ease SMA Severity in Mouse Models & Spinraza For 2 Years
We discuss how raising levels of Alpha-COP proteins, seems to ease SMA disease severity in mouse models. Also, SMA News Today forums moderator DeAnn Runge shares her perspective about being on Spinraza for 2 years, and also gives an update regarding her 9th Spinraza injection. Are you interested in understanding gene therapy? ExploreGeneTherapy.com has helpful information about gene therapy, including its history and how it is being investigated for the treatment of genetic diseases. Visit www.exploregenetherapy.com
19 Elo 20197min
