SMA Related Web Articles

These articles and their associated links are great sources of information for those of us with SMA

Avexis Announces Plan to Initiate Phase 1 Trial in SMA Type 2 Utilizing Intrathecal Delivery of AVXS-101

http://www.curesma.org/news/avxs-101-strong-trial.html?referrer=https://search.yahoo.com/


FDA Approves AveXis' Pivotal Trial of AVXS-101 For SMA Type 1

https://smanewstoday.com/2017/10/03/fda-approves-pivotal-trial-of-avxs-101-for-sma-type-1/


Study of Intrathecal Administration of AVXS-101 for SMA (STRONG)

https://www.clinicaltrials.gov/ct2/show/NCT03381729?term=AVXS-101&rank=1


Gene Replacement Therapy Clinical Trial for Patients With SMA Type 1 (STR1VE)

https://clinicaltrials.gov/ct2/show/NCT03306277


Biogen Unlikely To See Spinraza Rival In 2018 As AveXis Delayed

https://www.investors.com/news/technology/biogen-unlikely-to-see-spinraza-rival-in-2018-as-avexis-delayed/


Local family with little one battling Spinal Muscular Atrophy says "miracle drug" is changing their daughters life

http://fox4kc.com/2017/11/13/local-family-with-little-one-battling-spinal-muscular-atrophy-says-miracle-drug-is-changing-their-daughters-life/


Clinical Trials Bring Hope to Kids with Spinal Muscular Atrophy

https://directorsblog.nih.gov/2017/11/21/clinical-trials-bring-hope-to-kids-with-spinal-muscular-atrophy/


SMA Could Soon Be on Newborn Screening List in the U.S.

https://smanewstoday.com/2017/05/18/sma-could-soon-be-on-newborn-screening-list/


Moms push for newborn genetic screening so families avoid 'paralyzing grief'

https://www.today.com/parents/moms-advocate-genetic-screening-help-others-t114088


Spinal Muscular Atrophy - Global market size will increase 2 folds by 2025

http://www.satprnews.com/2017/11/08/spinal-muscular-atrophy-global-market-size-will-increase-2-folds-by-2025/


Young Man with Significant Disabilities Argues for Inclusion

https://www.thinkinclusive.us/significant-disabilities-inclusion/


This Man with Spinal Muscular Atrophy Has a Simple Way of Surviving Even Life's Biggest Challenges

http://blog.theautismsite.com/shane-burcaw/


Top trainee hopes unexpected discovery may help children with genetic disease

http://www.ottawahospital.on.ca/en/youre-in-my-care/top-trainee-hopes-unexpected-discovery-may-help-children-genetic-disease/

 

Two Good Options

https://www.genengnews.com/gen-exclusives/two-good-options/77900998


Two New Ways to Treat A Deadly Disease: Spinal Muscular Atrophy

http://blogs.plos.org/dnascience/2017/11/02/two-new-ways-to-treat-a-deadly-disease-spinal-muscular-atrophy/


Gene therapy's new hope: A neuron-targeting virus is saving infant lives

http://www.sciencemag.org/news/2017/11/gene-therapy-s-new-hope-neuron-targeting-virus-saving-infant-lives


Exosome-derived protein may serve as an effective biomarker for spinal muscular atropher

http://www.exosome-rna.com/exosome-derived-protein-may-serve-as-an-effective-biomarker-for-spinal-muscular-atrophy/


New hope for families affected by spinal muscular atrophy, with TGA approving drug

http://www.theage.com.au/national/new-hope-for-families-affected-by-spinal-muscular-atrophy-with-tga-approving-drug-20171104-gzet21.html/


Nusinersen versus Sham Control in Infatile-Onset Spinal Muscular Atrophy

http://www.nejm.org/doi/full/10.1056/NEJMoa1702752


AveXis Announces New England Journal of Medical Publication of Phase 1 Data of AVXS-101 Gene Replacement Therapy in Spinal Muscular Atrophy Type 1

http://biotech-365.com/avexis-announces-new-england-journal-of-medicine-publication-of-phase-1-data-of-avxs-101-gene-replacement-therapy-in-spinal-muscular-atrophy-type-1/


Gene replacement tx beneficial in spinal muscular atrophy

https://medicalxpress.com/news/2017-11-gene-tx-beneficial-spinal-muscular.html


Experimental baby gene therapy offers solution to spinal muscular atrophy

http://indianexpress.com/article/technology/science/experimental-baby-gene-therapy-offers-solution-to-spinal-muscular-atrophy-4919676/