Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for myotonic dystrophy type 1 (DM1), the most common adult-onset form of ...
Elevated levels of lipoprotein (a) is a genetic disease that causes cardiovascular disease. Steven Nissen, M.D., addresses why it’s important to find treatments for this genetic risk factor.
The genetic modification therapy market presents key opportunities in developing specialized treatments for rare, inherited, ...
Dentatorubral-pallidoluysian atrophy (DRPLA) is a lethal neurodegenerative disorder caused by pathogenic expansion of CAG repeats within the atrophin-1 (ATN1) gene. As DRPLA belongs to the broader ...
A new research paper was published in Volume 17, Issue 12 of Aging-US on December 1, 2025, titled “CD47 antisense ...
The n-Lorem Foundation and the EspeRare Foundation announced today a strategic collaboration to expand access in Europe to ...
Currently available therapies such as niacin and PCSK9 inhibitors such as Repatha (evolocumab) and Praluent (alirocumab) can reduce Lp (a) levels, but their impact is modest. Another challenge in ...
Wave expects to initiate a Phase 2a multidose portion of WVE-007 INLIGHT clinical trial in individuals living with obesity ...
Aperture Therapeutics is advancing APRTX-003, an RNA-based antisense therapy targeting MMP9, as a potential ALS treatment.
Pancreatic ductal adenocarcinoma (PDAC) is the most lethal form of pancreas cancer. It's also the most common form of the ...
Initial AX-0810 data show no safety signals after 4 weeks of dosing and pharmacokinetics consistent with non-clinical data; ...
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