Exicure’s gene c prolongs survival, increases the amount of SMN proteins, and shows limited toxicity in a mouse model of the disease, study data show. The investigational compound is a spherical nucleic acid (SNA) formulation of nusinersen, the active substance in Spinraza, made by Biogen and approved to treat all…
Decreased levels of an enzyme lead to cellular dysfunction and neuronal degeneration involved in the development of spinal muscular atrophy (SMA), a new mouse study suggests. The study, “Neuronal activity regulates DROSHA via autophagy in spinal muscular atrophy,” was published in Scientific Reports. An enzyme called DROSHA regulates the…
Targeted therapies or dietary supplements that work to improve the metabolism of certain molecules in muscle cells may slow the progression of spinal muscular atrophy (SMA), results of an early study in mice show. The study, “Interventions Targeting Glucocorticoid-Krüppel-like Factor 15-Branched-Chain Amino Acid Signaling Improve Disease Phenotypes…
AveXis is expanding its operations to North Carolina, opening up a new manufacturing center in Durham County, North Carolina Gov. Ray Cooper recently announced in a press release. The company is anticipated to invest about $55 million into the new facility and open up about 200 new jobs, giving…
Canada’s healthcare system is excellent for people with common ailments like diabetes or high blood pressure, but it’s “basically failing the nearly three million Canadians with rare diseases.” So says Durhane Wong-Rieger, president and CEO of the Canadian Organization for Rare Disorders (CORD), a Toronto-based network representing 102 patient advocacy…
Roche has stopped the development of olesoxime, a treatment candidate for spinal muscular atrophy (SMA) following disappointing 18-month results in an ongoing clinical study, the company announced. In a statement shared online through TreatSMA, Roche mentioned the most recent results of the OLEOS Phase 2 trial (…
As President Trump signed the recently passed Right to Try legislation into law in a White House ceremony, Jordan McLinn of Indianapolis tried twice to embrace him. The third time, 9-year-old Jordan finally got the hug he wanted — as well as a kiss on the forehead. The video of…
Scholar Rock announced it has completed dosing a first group of healthy volunteers in its Phase 1 clinical study of SRK-015, a spinal muscular atrophy (SMA) treatment candidate. SRK-105 selectively blocks the activation of myostatin, a growth factor produced primarily in skeletal muscle cells that helps to maintain a healthy muscle…
Envisagenics announced that it has been given a two-year, $1.5 million grant from the National Institutes of Health (NIH) to further advance SpliceCore, a new biomarker and drug discovery platform. The award, a Small Business Innovation Research (SBIR) Phase 2 grant, will help Envisagenics in further developing SpliceCore, a…
Should scientists have the right to edit the genes of future generations to eliminate hundreds, if not thousands, of potential rare diseases? Or should researchers restrict their use of genome editing to somatic cells, so that they don’t pass changes on to the next generation? What about stem-cell research, three-parent…
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