SMA Related Web Articles

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

Updates From Trial of AveXis SMA1 Gene Therapy Trial Show Impressive Results in Most Infants

https://smanewstoday.com/2017/04/25/sma1-gene-therapy-avxs-101-infants-clinical-trial/#at_pco=tst-1.0&at_si=5b0eb08821085b6e&at_ab=per-2&at_pos=1&at_tot=2


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/

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