Sentences with word «nusinersen»

A blind national clinical trial of nusinersen involving 121 SMA - 1 patients found that babies receiving the drug showed significant motor function improvement when compared with babies in the trial who didn't get the drug.

Biotechnology firms Biogen and Ionis Pharmaceuticals cut short a phase - 3 clinical trial for the investigational drug nusinersen as it met endpoint...

Mice with only nusinersen didn't gain much weight past 6 grams.

They report they have evidence that an experimental medicine aimed at this target works as a «booster» in conjunction with a drug called nusinersen that was recently FDA - approved to improve symptoms of the disorder in mice.

Before the FDA approved nusinersen, which is marketed by Biogen and Ionis Pharmaceuticals as Spinraza, the only care options were palliative care or supportive care, including physical therapy, respiratory help or assistive devices.

For now, they wrote, researchers and clinicians will continue to share any new data about nusinersen use in SMA patients to inform future decisions, including how broadly treatment should be pursued.

But both FDA and the European Medicines Agency have entered nusinersen in their fast track processes for drug approval.

«Our approach was to work with those that developed nusinersen to identify a way to boost their drug's effects on the sickest of the sick.»

Even if the final results show nusinersen doesn't work as well as hoped, there is still cause for optimism.

The Johns Hopkins researchers and their colleagues say the «boosted» nusinersen therapy, if confirmed by further studies, has the potential to significantly improve the lives of people with spinal muscular atrophy, some with forms of the disease with only weeks or months to live, and others who are unable to sit, stand or walk.

Relief could also come as new treatments — and resulting market competition for nusinersen — emerge, Burgart said.

Two recent, late - stage clinical trials were stopped because nusinersen, developed by Ionis of Carlsbad, California, and now licensed to Biogen of Cambridge, Massachusetts, was so effective in restoring motor function in children and babies that it was deemed unethical to deny the drug to children in the placebo arms of the trials.

And this time, too, it turned out that the drug with the active substance nusinersen, which partly compensates for an inherited genetic error, altered the children's condition.

To receive nusinersen, the babies had to have developed SMA symptoms between 3 weeks and 6 months of age; the drug was injected into the cerebrospinal fluid surrounding the spinal cord.

Hua, now at the Institute of Neuroscience at Soochow University in Jiangsu, China, led the initial work at Cold Spring Harbor Laboratory on developing the drug and is named as co-inventor on nusinersen's patent.

In their November announcement about the second trial's halt, Ionis and Biogen, which has licensed nusinersen and has dubbed it Spinraza, said the treated children experienced a «highly statistically significant improvement» in motor function compared to untreated children.

It's a positive sign that spinal cord injections of nusinersen, the newly approved drug, restore some motor function and prolong survival by improving SMN production, Hart said, but researchers can make further and perhaps more lasting headway by understanding how the lack of SMN ultimately undermines muscle function.

An antisense - based treatment called nusinersen, which removes a roadblock in the expression of the second SMN gene, was recently approved by the FDA.

«The new FDA - approved drug nusinersen may have a «ceiling effect,» in that it may not completely treat the disorder,» says Charlotte Sumner, M.D., associate professor of neurology at the Johns Hopkins University School of Medicine.

SPINRAZA ® (nusinersen) has been approved in global markets for the treatment of spinal muscular atrophy (SMA).

Mice with spinal muscular atrophy were injected with ASO - A alone, a low 50 milligram per kilogram dose of nusinersen alone, both ASO - A and nusinersen, or saline at day one after birth.

Yet a few parents have posted videos online showing children given the drug, called nusinersen, who appear to be sitting and even walking with assistance.

But the results of their study, they add, offer at least one path to extending the usefulness of nusinersen.

The trial of nusinersen was stopped in August when it became clear it was effective, making it unethical not to give the real drug to those on the placebo.

Animal studies, and postmortems of children who died despite being given nusinersen, show widespread distribution of the antisense molecule in the brain and spinal cord, says Muntoni, who has helped develop and test therapies such as nusinersen.

Biogen, which makes nusinersen, has set the price per injection of nusinersen at $ 125,000, or about $ 750,000 for the first year of treatment.

The first and newest FDA - approved treatment of nusinersen doesn't completely cure the disease, so Sumner's research team wanted to see if using ASO - A in conjunction with nusinersen would further improve muscle form and function in mice with spinal muscular atrophy.

Saline and ASO - A mice lived an average of 18 days, and nusinersen only - treated mice lived an average of 25 days.

The U.S. Food and Drug Administration approved the SMA drug, nusinersen, later that year.

Pediatric anesthesiologist and bioethicist Alyssa Burgart holds a vial of the drug nusinersen, which has been approved for people with spinal muscular atrophy, but carries a hefty price tag.

Chief among those concerns is that the $ 125,000 - per - dose cost of the drug, nusinersen, could restrict long - term patient access to it and the ability of clinics to provide it.

Neuroscientists are optimistic that the success of nusinersen may portend similar positive results for antisense therapies now being developed to fight other genetically linked nervous system diseases like amyotrophic lateral sclerosis and Huntington disease.

The recent approval of SpinrazaTM (nusinersen), jointly developed by Ionis Pharmaceuticals and Biogen, marks the arrival of a new class of biological products — oligonucleotide therapeutics.

Hua's extensive work in Krainer's lab, in collaboration with Ionis, led to the identification and preclinical development of nusinersen, a snippet of modified RNA called an antisense oligonucleotide that intervenes in the splicing process to ensure that SMN2 expresses the full - length SMN protein.

* Correction, 24 February 2017, 2:09 p.m.: Because of an editing mistake, the contributions of Yimin Hua to the development of nusinersen were removed from the original version of this story.

Still, when the brain cells and spinal cord cells of these babies were examined at autopsy, there was clear evidence that nusinersen had tricked SMN2 into producing a great deal more of the full length, motor neuron - protecting protein: two to six times more copies of SMN's messenger RNA were found in spinal cord samples from nusinersen - treated babies than in autopsy samples from untreated infants.

On 7 November, a trial of the drug, nusinersen, in wheelchair - bound children aged 2 to 12, was stopped on the grounds that it was unethical to deny the drug to children in the control arm, given the positive results in the treated children.

* Update, 23 December: FDA today approved the use of nusinersen to treat spinal muscular atrophy.

And today, a paper appearing in The Lancet provides compelling biological evidence that nusinersen is having its desired effect in the cells of the brain and spinal cord.

Results from a phase 1 study of nusinersen (ISIS - SMNRx) in children with spinal muscular atrophy.