https://www.ajmc.com/view/researchers-outline-promising-future-for-sma-dmd-with-emergence-of-gene-therapies-asos
Researchers Outline Promising Future for SMA, DMD With Emergence of Gene Therapies, ASOs
September 12, 2020
www.SMATreatmentReport.com
https://www.ajmc.com/view/researchers-outline-promising-future-for-sma-dmd-with-emergence-of-gene-therapies-asos
Researchers Outline Promising Future for SMA, DMD With Emergence of Gene Therapies, ASOs
September 12, 2020
https://www.ajmc.com/view/improved-treatment-options-for-sma-present-new-opportunities-challenges
Improved Treatment Options for SMA Present New Opportunities, Challenges
September 5, 2020
Laura Joszt
https://investors.scholarrock.com/news-releases/news-release-details/scholar-rock-announces-srk-015-has-received-rare-pediatric
Scholar Rock Announces that SRK-015 has Received Rare Pediatric Disease Designation from U.S. FDA for the Treatment of Spinal Muscular Atrophy
B.C. baby randomly selected by drug company to receive life-changing $3 million treatment
B.C. baby randomly selected by drug company to receive life-changing $3 million treatment
By Amy Judd Global News
Posted August 13, 2020 7:30 pm
https://www.fda.gov/news-events/press-announcements/fda-approves-oral-treatment-spinal-muscular-atrophy
FDA Approves Oral Treatment for Spinal Muscular Atrophy
Evrysdi (risdiplam)
https://www.prweb.com/releases/catalent_gene_therapy_facility_receives_fda_approval_as_an_additional_manufacturing_site_for_avexis_gene_therapy/prweb17307018.htm
Catalent Gene Therapy Facility Receives FDA Approval as an Additional Manufacturing Site for AveXis’ Gene Therapy
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Catalent, the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, cell and gene therapies, and consumer health products, today announced that it was approved by the U.S. Food and Drug Administration (FDA) to produce commercial drug substance intermediate for AveXis’ spinal muscular atrophy (SMA) gene therapy at its manufacturing facility located in Harmans, Maryland.
https://www.pharmacytimes.com/news/study-patients-with-pre-symptomatic-spinal-muscular-atrophy-demonstrate-sustained-benefit-from-nusinersen
Study: Patients with Pre-Symptomatic Spinal Muscular Atrophy Demonstrate Sustained Benefit from Nusinersen
https://www.globenewswire.com/news-release/2020/06/10/2046158/0/en/New-Results-From-Landmark-NURTURE-Study-Show-That-Pre-Symptomatic-SMA-Patients-Treated-With-SPINRAZA-nusinersen-Continue-to-Demonstrate-Sustained-Benefit-From-Treatment.html
New Results From Landmark NURTURE Study Show That Pre-Symptomatic SMA Patients Treated With SPINRAZA® (nusinersen) Continue to Demonstrate Sustained Benefit From Treatment
https://www.prnewswire.com/news-releases/cure-sma-announces-new-funding-for-network-of-prominent-sma-clinical-research-and-treatment-centers-301070498.html
Cure SMA Announces New Funding For Network Of Prominent SMA Clinical Research And Treatment Centers
The Pediatric Neuromuscular Clinical Research Network Will Continue to Drive Treatment Development Under Cure SMA’s Funding
FDA grants Orphan Drug status for Shift’s lead compound for SMA
FDA grants Orphan Drug status for Shift’s lead compound for SMA
May 14, 2020
Steve Oconnor
Shift’s E1V1.11 drug compound to treat Spinal Muscular Atrophy (SMA) granted Orphan Drug Status by FDA.
Overland Park: The Food and Drug Administration (FDA) granted orphan drug designation for Shift Pharmaceutical’s lead drug compound to treat all forms of spinal muscular atrophy (SMA). Orphan Drug Status provides incentives for companies to research drug developments to treat rare diseases such as SMA.
https://finance.yahoo.com/news/roche-provides-regulatory-risdiplam-treatment-200500098.html
Roche provides regulatory update on risdiplam for the treatment of spinal muscular atrophy (SMA)
https://www.fiercepharma.com/pharma/should-biogen-s-spinraza-for-sma-be-delayed-amid-covid-19-no-say-these-parents
Should Biogen’s Spinraza for SMA be delayed amid COVID-19? No, say these parents