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1 week ago | 
fapnewstoday.com | Margarida Maia |Steve Bryson |Katherine Poinsatte
Here in New Zealand, clinicians and advocates are pushing our country’s government to update its genetic editing and modification legislation. Historically, New Zealand has maintained some of the strictest gene technology regulations globally. Currently, genetic modification is heavily regulated under the . The legislation was intended, in part, to protect the environment and public health from genetically modified organisms, which the act classifies as new organisms.
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1 week ago | 
alsnewstoday.com | Margarida Maia |Kristin Neva |Katherine Poinsatte
Neurosense Therapeutics’ oral therapy PrimeC significantly reduces blood levels of miRNAs — molecules involved in regulating gene activity — associated with disease progression and survival in people with amyotrophic lateral sclerosis (ALS). That’s according to six-month biomarker data from the Phase 2b PARADIGM clinical trial (NCT05357950), in which 68 adults with ALS were randomly assigned to receive either PrimeC or a placebo for six months, followed by one year on the therapy.
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1 week ago | 
cysticfibrosisnewstoday.com | Margarida Maia |Marisa Wexler |Andrea Lobo |Katherine Poinsatte
Pregnancy does not negatively impact survival or lung function in women with cystic fibrosis (CF) and, in fact, may be associated with a lower risk of death, according to a U.S. study that compared CF women who had been pregnant with those who had never been pregnant. No significant differences in survival were found among the two groups by the research team, from Stanford University School of Medicine and Stanford Health Care, both in California, who tracked data spanning 20 years.
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1 week ago | 
smanewstoday.com | Steve Bryson |Katherine Poinsatte |Marta C. Figueiredo |Sherry Toh
Three men with late-onset spinal muscular atrophy (SMA) undergoing treatment with the approved therapy Evrysdi (risdiplam) were able to conceive with their partners, a case series reported. None of the pregnancies were planned, and the researchers noted there were no missed treatment doses. Among the three, one pregnancy was voluntarily terminated, while the other two men’s partners gave birth to healthy babies.
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1 week ago | 
sjogrenssyndromenews.com | Lindsey Shapiro |Katherine Poinsatte |Marisa Wexler
A naturally occurring molecule that has the ability to regulate gene activity may represent a promising therapeutic target for Sjögren’s disease and other autoimmune conditions, according to a study. Mimicking the activity of the molecule, miR-216a-3p, eased inflammation and scarring in a rat model of the disease.
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3 weeks ago | 
cysticfibrosisnewstoday.com | Margarida Maia |Andrea Lobo |Patricia Inacio |Katherine Poinsatte
Trikafta (elexacaftor/tezacaftor/ivacaftor) improves lung function and nutrition status in adults and adolescents with cystic fibrosis (CF), and appears to work particularly well in those with more severe lung disease, according to a real-world study from the U.S.Researchers also observed that Trikafta appears to work about as well for patients who carry one or two copies of F508del in the CFTR gene, the most common CF-causing mutation.
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3 weeks ago | 
smanewstoday.com | Lindsey Shapiro |Steve Bryson |Katherine Poinsatte
The balance of amino acids capable of influencing neurological function is disrupted in patients with spinal muscular atrophy (SMA) and in mouse models, a study found. Observed changes in patients were partially normalized after treatment with the disease-modifying therapy Spinraza (nusinersen). Treatment with one of the amino acids, D-serine, improved motor function in a mouse model.
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3 weeks ago | 
liverdiseasenews.com | Marisa Wexler |Katherine Poinsatte |Sophie Bourton |Lindsey Shapiro
Analyzing the presence of different proteins in liver tissue under a microscope can be used to help diagnose progressive familial intrahepatic cholestasis (PFIC) and distinguish between its different types, according to a study done in India. The findings indicate that this type of relatively cheap and accessible analysis, called immunohistochemistry, could be used to aid in identifying and discriminating PFIC types when standard — but more expensive — genetic testing isn’t an option.
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3 weeks ago | 
pnhnews.com | Patricia Inacio |Katherine Poinsatte |Andrea Lobo |Brandi Lewis
Paroxysmal nocturnal hemoglobinuria (PNH) was identified as the likely cause of a stroke in a young man, according to a report from India.
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4 weeks ago | 
smanewstoday.com | Marisa Wexler |Steve Bryson |Katherine Poinsatte
It’s been almost six years since the one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) was first approved in the U.S. to treat young children with spinal muscular atrophy (SMA). Now, more than 95% of babies diagnosed with SMA are being treated with Zolgensma, according to Daniel Grant, vice president and global program head at Novartis, the company that markets the therapy.
