CHICAGO, Illinois — A genetic change or variant in a gene called SCN2A is a known cause of infantile seizures, autism spectrum disorder and intellectual disability, as well as a wide range of other moderate-to-profound impairments in mobility, communication, eating and vision. The severity of these disorders can vary widely...
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South Bend, Indiana – What could cancer teach us about tuberculosis? That’s a question Meenal Datta has been chasing since she was a graduate student. Once the body’s immune system is infected with tuberculosis, it forms granulomas — tight clusters of white blood cells — in an attempt to wall...
Notre Dame’s famous “Touchdown Jesus” mural lit up for Rare Disease Day on Tuesday.
FALLS CHURCH, Va. – This year, nearly 2 million Americans will be diagnosed with cancer. But one-quarter won’t fill their expensive prescriptions, and another 20% will ration their drugs due to cost – which can total $15,000 per month or more. But thanks to a partnership between NOVA ScriptsCentral and...
Authorization to start clinical trial in the US granted for rare paediatric bone disease Mont-Saint-Guibert – Novadip Biosciences (“Novadip”), a clinical stage company developing treatments to regenerate impaired tissues in patients with significant unmet medical needs, today announced that the U.S. Food and Drug Administration (FDA) has approved the Company’s...
EAST HANOVER, N.J. – Novartis today announced important findings from a real-world study evaluating the safety and efficacy of alpelisib for people living with PIK3CA-Related Overgrowth Spectrum (PROS) who received treatment daily for at least 24 weeks. Results from EPIK-P1 showed alpelisib effectively reduced volume of clinically significant PROS-related lesions and...
Basel – Novartis today announced Phase II primary endpoint data showing investigational iptacopan (LNP023) – a first-in-class, oral, targeted factor B inhibitor – reduced protein in the urine (proteinuria), an increasingly recognized surrogate marker correlating with progression to kidney failure[2], and showed promise in stabilizing kidney function in patients with...
Basel – Novartis announced that the Novartis Vaccines Institute for Global Health (NVGH) has been awarded a grant from the Wellcome Trust to develop a bivalent vaccine for Typhoid fever, a disease that affects more than 21 million people worldwide every year. The Euro 5.15 million grant will fund preclinical...
Novartis Entresto® granted expanded indication in chronic heart failure by FDA Entresto is the first and only therapy approved in the US to treat patients diagnosed with guideline-defined heart failure to include both those with heart failure with reduced ejection fraction (HFrEF) and many with heart failure with preserved ejection...
LEXINGTON, Mass. — Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today announced a strategic collaboration and capsid license agreement with Novartis Pharma AG, a subsidiary of Novartis AG (NYSE: NVS) to advance potential gene therapies for Huntington’s disease (HD) and spinal muscular atrophy (SMA)....