New Data about Spinraza® in Infants with Spinal Muscular Atrophy Has Been Shared
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New Data about Spinraza® in Infants with Spinal Muscular Atrophy Has Been Shared

Biogen Inc. has provided updates on their research into Spinraza® (nusinersen) in infants with spinal muscular atrophy. At the moment, Sprinraza is the only approved treatment for the condition. Data from…

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Data from Studies of a Potential Drug for Spinal Muscular Atrophy Show Very Encouraging Results
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Data from Studies of a Potential Drug for Spinal Muscular Atrophy Show Very Encouraging Results

Encouraging interim data has been shared from two studies of risdiplam (RG7916), a drug being researched as a potential treatment for spinal muscular atrophy types 1, 2, and 3. For…

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Researchers Find a New Way to Deliver Nusinersen to Patients with Spinal Muscular Atrophy
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Researchers Find a New Way to Deliver Nusinersen to Patients with Spinal Muscular Atrophy

A recent study suggests an alternative procedure for delivering the drug nusinersen to patients being treated for spinal muscular atrophy. Although more research needs to be done to estabilish its…

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So Why DOES Canada have Scarcer Access to Rare Disease Therapies?
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So Why DOES Canada have Scarcer Access to Rare Disease Therapies?

According to a story from CheckOrphan, Durhane Wong-Rieger, the CEO of the Canadian Organization for Rare Disorders, says that the Canadian health system has been letting down the roughly three…

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A Man With Spinal Muscular Atrophy is Stuck Without Access to Costly Treatment
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A Man With Spinal Muscular Atrophy is Stuck Without Access to Costly Treatment

According to a story from cbc.ca, Cole Pringle is a 31-year-old man from Regina, Saskatchewan with spinal muscular atrophy, a debilitating illness that severely affects his mobility. He was diagnosed…

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Researchers are Investigating the Link Between Circadian Rhythms and Spinal Muscular Atrophy
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Researchers are Investigating the Link Between Circadian Rhythms and Spinal Muscular Atrophy

A recent study has found that ‘clock’ genes that help to regulate the body’s circadian rhythm may be affected by spinal muscular atrophy. The original paper, which is published in…

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Results From a Phase II Trial of Reldesemtiv For Treating Spinal Muscular Atrophy Have Been Announced
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Results From a Phase II Trial of Reldesemtiv For Treating Spinal Muscular Atrophy Have Been Announced

Data from a Phase II clinical trial of the experimental treatment reldesemtiv for the treatment of spinal muscular atrophy has been announced. The drug has shown positive effects in the…

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ICYMI: Experimental Drug for Spinal Muscular Atrophy Gets Orphan Drug Designation
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ICYMI: Experimental Drug for Spinal Muscular Atrophy Gets Orphan Drug Designation

According to a story from BioSpace, the pharmaceutical company Scholar Rock recently announced that its drug candidate SRK-015 was recently granted Orphan Drug Designation by the U.S. FDA. The drug…

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Australian Boy With Spinal Muscular Atrophy is Now Able to Get Critical Treatment
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Australian Boy With Spinal Muscular Atrophy is Now Able to Get Critical Treatment

According to a story from the Sydney Morning Herald, two-year-old William McLennan, son of Naomi Taylor and Ben McLennan, was diagnosed with spinal muscular atrophy about a year ago. The…

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Parents of Kids With Spinal Muscular Atrophy Are Worried About Signing Them up for Clinical Trials
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Parents of Kids With Spinal Muscular Atrophy Are Worried About Signing Them up for Clinical Trials

According to a story from SMA News Today, a recent study has demonstrated that the parents of children with spinal muscular atrophy are often skeptical about enrolling their kids in…

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Investigational Gene Therapy for Spinal Muscular Atrophy Gets SAKIGAKE Designation
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Investigational Gene Therapy for Spinal Muscular Atrophy Gets SAKIGAKE Designation

According to a story from GlobeNewswire, The pharmaceutical company AveXis, Inc., a company that specializes in the development of gene therapies, recently announced that its candidate AVXS-101 was awarded SAKIGAKE…

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The FDA is Beginning to Accept New Endpoints and “Real World” Data in Clinical Trials
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The FDA is Beginning to Accept New Endpoints and “Real World” Data in Clinical Trials

According to a story from Charcot-Marie-Tooth Disease News, the U.S. Food and Drug Administration (FDA), under the leadership of commissioner Scott Gottlieb, has begun to change clinical trials by accepting…

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Clinic Beats Slow Hospitals to be the First to Deliver Spinal Muscular Atrophy Drug in Michigan
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Clinic Beats Slow Hospitals to be the First to Deliver Spinal Muscular Atrophy Drug in Michigan

The day after Christmas in 2016 brought a post-Holiday surprise to many in the U.S. with spinal muscular atrophy. December 26, 2016 marked the day that Spinraza was approved by…

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