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…
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…
According to a story from kentlive.news, Lucy Frost of Tumbridge Wells is doing everything she can in an attempt to get the NHS to provide her son with the medication he…
According to the Santa Cruz Sentinel, Josalyn Catarino is an 11-year-old who loves Disneyland, TV romance shows, the ocean, and getting her fingernails painted. This, of course, sounds like any…
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…
Let's take a moment during these dog days of summer to recognize the SMA community during SMA awareness month! Spinal muscular atrophy (SMA) is a rare genetic disorder that causes…
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…
Asher Camp is five years old. His parents, Amanda and Jeremy, had two daughters before Asher. That’s why when Asher progressed differently, they knew to be concerned. After quick action,…
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…
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…
According to Medical Xpress, scientists have pinpointed how a potentially new treatment effectively targets spinal muscular atrophy (SMA). The results from the research could lead to further development in SMA…
Spinal muscular atrophy is a genetic disease with life threatening implications. In order to create effective therapies, doctors need the ability to target the root genetic mutation and stop it.…
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…
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…
The first patient has just been dosed in a Phase 3 trial of a drug to treat spinal muscular atrophy, reports Benzinga. The drug, called AVXS-101, is a gene therapy…
In an $8.7 billion agreement, Swiss drugmaker Novartis recently acquired AveXis. The company moved in such a way in order to acquire a developmental therapy for spinal muscular atrophy. The…
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…
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…
Spinal muscular atrophy is both rare and deadly. It’s uncommon enough that most people never worry about it-- but the consequences that happen if it goes undetected are devastating. That’s…
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…
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…
According to a story from macleans.ca, eight-year-old Mason Stewart has been dealing with the effects of spinal muscular atrophy since he began to walk; he developed an unusual walking gait…
Meisyn Ellison, a 13-year-old in Utah, adopted from China, battles the debilitating effects of her rare disease and now seeks to find her biological parents in hopes to give doctors…
The Lewis family has lived through it all. Their first born was diagnosed with spinal muscular atrophy (SMA) and did not live long, having passed away in 2013, reports Deseret News. And…
One of the most impressive moments of childhood is when a young child takes his or her first steps. Natalie Essex, age four, loves to walk. For Natalie, spinal muscular…
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