An HIV-derived nucleoside therapy now treats rare genetic diseases by restoring mitochondrial DNA and improving muscle ...
The n-Lorem Foundation and the EspeRare Foundation announced today a strategic collaboration to expand access in Europe to n-Lorem's antisense oligonucleotide (ASO) therapies for patients with rare ...
PacBio (NASDAQ: PACB), developer of the world's most advanced sequencing technologies, today announced plans to pursue a strategic collaboration with the n-Lorem Foundation and EspeRare that we ...
Noonan Syndrome, identified by Dr. Jacqueline A. Noonan, involves congenital heart defects and is linked to PTPN11 gene mutations. Cronkhite-Canada Syndrome, discovered by Dr. Wilma Jeanne Canada, is ...
While genetic testing may be diagnostic, ordering the tests requires physicians to consider the possibility of the illness first. Treatments Curative treatments are rare but available for diseases ...
Johns Hopkins Medicine laboratory scientists say they have developed a potential new way to treat a variety of rare genetic diseases marked by too low levels of specific cellular proteins. To boost ...
| Servier is committing to rare neurology for the long term, using focused science and strategic collaborations to bring new ...
CNBC's Squawk Box host Becky Quick opened up about her daughter Kaylie's battle with SYNGAP1, a rare genetic disorder ...
As genetic sequencing becomes more advanced and widely used, researchers expect to uncover more rare blood types. Each discovery expands our understanding of human variation and raises fresh ...
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