European Commission Grants Conditional Approval for Spinal Muscular Atrophy Treatment

Zolgensma, a gene therapy for spinal muscular atrophy (SMA), has recently been granted conditional approval by the European Commission (EC). This is extremely exciting news for those living with SMA…

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RNA Therapy Holds Promise for Treating SMA and Other Rare Diseases
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RNA Therapy Holds Promise for Treating SMA and Other Rare Diseases

by Jodee Redmond from In the Cloud Copy   Emma Larson’s parents were not particularly concerned when their daughter wasn’t standing or walking by her first birthday. They figured that plenty of children had not reached…

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Man Runs 400 Laps Around His Own House to Help Spinal Muscular Atrophy Patient
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Man Runs 400 Laps Around His Own House to Help Spinal Muscular Atrophy Patient

According to a story from The Charlotte Weekly, local resident Kevin Tobin recently ran 400 laps around his house---the equivalent of a marathon---to help raise money for Dan Donoher, a…

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“The Toddler that Doesn’t Toddle:” Raising a Child with Spinal Muscular Atrophy
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“The Toddler that Doesn’t Toddle:” Raising a Child with Spinal Muscular Atrophy

  As initially covered by MyLondon News, the family of a London boy with spinal muscular atrophy (SMA) is looking to advocate for others with this genetic disorder. Additionally, they…

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Long Term Data Looks Good for Spinal Muscular Atrophy Gene Therapy Zolgensma
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Long Term Data Looks Good for Spinal Muscular Atrophy Gene Therapy Zolgensma

According to a story from Benzinga, the Novartis Company AveXis recently announced the results of long term studies testing the impact of Zolgensma, a gene therapy treatment that was approved…

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The European Parliament is Relaunching their Network for Rare Diseases

The European Parliament has announced that they will be relaunching their "Network of Parliamentary Advocates for Rare Diseases." Its aim is to create a new policy framework which can improve…

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A College Student Started a Business to Inspire Others with Spinal Muscular Atrophy
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A College Student Started a Business to Inspire Others with Spinal Muscular Atrophy

  One of the wealthy regulars on the TV show Shark Tank made his millions by starting a home-based business selling tee shirts. An article in Bridgeport’s ctPost publication chronicles…

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Interview with Dr. Michelle Krishnan: New Therapies for Neurodevelopmental Disorders
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Interview with Dr. Michelle Krishnan: New Therapies for Neurodevelopmental Disorders

Front Line Genomics has recently interviewed Dr. Michelle Krishnan, who is the Translational Medicine Leader in Rare Diseases at Roche. She focuses on rare neurodevelopmental disorders, in which she works…

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New Oral Drug for SMA may Help Young Children with the Most Severe Form of the Disease
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New Oral Drug for SMA may Help Young Children with the Most Severe Form of the Disease

Spinal muscular atrophy (SMA) currently has two approved treatment options. Just 3.5 years ago this number was zero and in another year or so, researchers are hoping to increase it to…

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ICYMI: The Manufacturer of Spinal Muscular Atrophy Drug Zolgensma has Made a Lottery for Life
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ICYMI: The Manufacturer of Spinal Muscular Atrophy Drug Zolgensma has Made a Lottery for Life

As originally reported in Euronews, there is limited supply of expensive medicines for rare diseases, so it can be difficult to prioritize which patients will have their lives saved when…

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Drugmaker Novartis Launches Lottery for Access to Critical Spinal Muscular Atrophy Treatment
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Drugmaker Novartis Launches Lottery for Access to Critical Spinal Muscular Atrophy Treatment

According to a story from euronews.com, the drug maker Novartis has announced that it will give away its breakthrough spinal muscular atrophy gene therapy drug Zolgensma to 100 patients each…

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Parents React to Novartis’ International “Baby Lottery” for Zolgensma with Hope But Also With Outrage

According to a recent article in Euronews, this year one hundred children who were diagnosed with spinal muscular atrophy (SMA) will receive Zolgensma, the world’s highest-priced drug, at no cost.…

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Preliminary Findings From Spinal Muscular Atrophy Trial Recently Announced
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Preliminary Findings From Spinal Muscular Atrophy Trial Recently Announced

According to a story from gurufocus.com, the biopharmaceutical company Scholar Rock has recently announced the release of preliminary results from a phase 2 clinical trial. This clinical trial is testing…

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Catalyst is Meeting with the FDA to Discuss the Future of Firdapse for Congenital Myasthenic Syndromes

Firdapse, or amifampridine phosphate, is a medication currently approved for Lambert-Eaton myasthenic syndrome, or LEMS. However, Catalyst Pharmaceuticals has been working to evaluate if it can aid other neurological and…

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Queensland Family Calls for Spinal Muscular Atrophy Newborn Screening After Their Daughter was Diagnosed
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Queensland Family Calls for Spinal Muscular Atrophy Newborn Screening After Their Daughter was Diagnosed

According to a story from abc.net.au, parents Kellee and Jamie Clarkson of Queensland, Australia are calling for the implementation of newborn screening in the state for the rare disorder spinal…

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Data Indicates That Gene Therapy for Spinal Muscular Atrophy Could be Useful for Older Patients

According to a story from MedCity News, interim data from a phase 1/2 clinical trial testing Zolgensma, a gene therapy for spinal muscular atrophy, in patients between the ages of…

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