FA is a progressive neuromuscular disease that causes loss of coordination and mobility. My parents got my test results the ...
Medical Xpress on MSN1d
Little-studied RNA might be key to regulating genetic disorders like epilepsy and autism"The unique mechanism we've identified here suggests that there are more long non-coding RNAs underlying rare genetic ...
New research demonstrates the potential of precise genome editing technologies, called adenine base editors, to correct disease-causing mutations in stem cells from patients with X-linked chronic ...
At #STATSummit, three parents discussed the challenges they've faced trying to find treatments for their children with rare ...
Nager syndrome (NS) is an extremely rare disease that causes developmental problems and anomalies in facial bone structures and limbs. While the causative gene is known, its underlying mechanisms ...
Savara Inc. (Nasdaq: SVRA) (the Company), a clinical stage biopharmaceutical company focused on rare respiratory diseases, ...
A TEEN has spent the last 10 years in a wheelchair after he was diagnosed with a rare genetic muscular disease known as ...
After "coming out of the fog" post-diagnosis, Whitrod immersed herself in research, making connections with other families whose children were also living with rare genetic diseases. One of those ...
Researchers found that ultra-rare genetic variants play a significant role in the heritability of coronary artery disease ...
Emma Austin needs $172,000 therapy every two weeks to slow the progression of Batten disease, which has "no known survivors." ...
Leber's congenital amaurosis is a rare genetic disease caused by mutations in genes. Frida Bollani Magoni, daughter of ...
When a gene produces too much protein, it can have devastating consequences on brain development and function. Patients with ...
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