Sma News & Perspectives

We discuss how the National Center For Advancing Translational Studies, (NCATS), warns that rare diseases constitute a public health issue. Also, SMA News Today forums moderator DeAnn Runge shares her perspective about going with the flow and living 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

We discuss a recent study that suggests that Spinraza is safe and effective in adults with SMA. Also, Community Development Manager Kevin Schaefer reads from his latest column, in which he recounts a negative experience with flying. 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

SMA News Today’s Director of Multichannel Content, Michael Morale, discusses a rare case of severe SMA in India, that was partly evident by bone fractures at birth. Also, Community Development Manager Kevin Schaefer talks about his experiences at the 2019 Cure SMA conference. Cure SMA is the largest SMA nonprofit organization, and this year’s conference took place in Anaheim, California. 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

We discuss how Risdiplam is showing promise for treating SMA Types 2 and 3. Also, SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how airline companies, and the FAA, refuse to make it easier for those of us in wheelchairs to have the ability to fly like the able-bodied. 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

We discuss how Risdiplam improves survival and motor functions in infants with Type 1 SMA. Also, Columnist Michael Casten reflects on his daughter's ninth birthday, and how far she's come since her initial SMA diagnosis. 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

SMA News Today’s Director of Multichannel Content, Michael Morale, discusses a study that states that SBMA patients are more likely to be affected by metabolic disorders, including heart and liver disease. Also, Columnist Alyssa Silva writes about how spontaneous adventures to faraway coffee shops help her celebrate the good days 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

In episode 37 of the SMA News Today Podcast, host Kevin Schaefer interviews Courtney Smith and her boyfriend Trevor Rogerson. They live in Seattle, Washington, and Courtney has SMA Type 2. 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

We discuss how Zolgensma continues to extend survival in SMA Type 1 patients several years after dosing. Also, SMA News Today forums moderator DeAnn Runge shares her perspective with regards to how she sees SMA changing in the future, for those of us who suffer from this disease. 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

We discuss how SMA patients in Saskatchewan, Canada, have gained wider access to Spinraza. Also, Community Development Manager Kevin Schaefer talks about recognizing his limitations in his latest column. 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

SMA News Today’s Director of Multichannel Content, Michael Morale, discusses a long-term trial that states how Spinraza was found to improve and restore motor function in children with late onset SMA. Also, Community Development Manager Kevin Schaefer talks about Kevan Chandler's latest book, which is in stores now. Chandler is an author and nonprofit founder, who has 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

We discuss a study that suggests how high intensity training improves fitness while being safe for patients with spinal bulbar muscular atrophy (SBMA). Also, SMA News Today’s Director of Multichannel Content, Michael Morale, discusses 4 tips that can make your upcoming Spinraza treatments easier. 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

We discuss a recent study that suggests how parents need supportive care as their children transition to wheelchairs. Also, Community Development Manager Kevin Schaefer talks about how Spinraza has affected his head and neck control in his latest column. 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

SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how some Spinraza users shared their stories in a Biogen webcast on how they went from a gut wrenching diagnosis to a wonderful drug. Also, Community Development Manager Kevin Schaefer reads from a forum topic about swimming pool lifts for people with disabilities. 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

In episode 36 of the SMA News Today Podcast, hosts Kevin Schaefer and Michael Morale discuss the FDA-approval of AveXis’ drug Zolgensma, and related news stories. We also look at some of our recent columns and forum posts, and share our thoughts. Listen here, and be sure to subscribe to the podcast. 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

We discuss how a nonprofit group is working to raise rare disease awareness in India. Also, SMA News Today forums moderator DeAnn Runge shares her perspective regarding how living with SMA has taught her the meaning of patience. 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

We discuss a recent mouse study that explores the underlying cause of motor neuron degeneration in SBMA. Also, Community Development Manager Kevin Schaefer talks about finding the sweet spot, when it comes to getting blood drawn and positioning in his wheelchair. 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

SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how Patient Airlift Services (PALS) uplifts the spirits of rare disease patients by offering free flights. Also, Community Development Manager Kevin Schaefer talks about how his head and neck control have improved since he started receiving Spinraza. 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

We discuss how ZOLGENSMA is helping treated babies to breathe and sit on their own. Also, SMA News Today’s Director of Multichannel Content, Michael Morale, discusses how his best friend inspired him to continue to improve and enrich his life. 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

We discuss a recent study that suggests how certain changes in bladder and the colon, may underlie gastrointestinal issues in SMA. Also, Community Development Manager Kevin Schaefer reads from his latest column about what medical advancements mean for adults 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

SMA News Today’s Director of Multichannel Content, Michael Morale, discusses a potential relationship that was found between early non-neuromuscular symptoms and the lack of SMN protein in SMA patients. Also, Community Development Manager Kevin Schaefer reads from a recent post in the SMA News Today forum regarding the recent FDA-approval of Avexis' Zolgensma, and we explain the key differences between this drug and Spinraza. 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