NAF has launched a petition on Change.org calling on the FDA to prioritize treatment options for rare diseases with urgent unmet needs, including Spinocerebellar Ataxia (SCA).
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How does a medical drug get to patients? Research is being done every day to discover new or better ways to treat diseases and various medical conditions. In order to determine if these treatments will help patients, studies known as “clinical trials” need to be done before these methods of Read More…
Antisense Oligonucleotides (also known as ASOs or AONs) are small molecules that can be used to prevent or alter the production of proteins. Proteins are the workforce of the cell, taking care of most cellular processes. They are generally made in a two-step process: first, a specific protein-coding gene is Read More…
One of the most important features of neurons (Purkinje cells, for example), is that they are capable of electrical communication. Think of the last time you saw a TV intro or movie montage with a depiction of the brain on a microscopic level – though it’s technically invisible to the Read More…
The information that allows the normal development and functioning of each human being is coded in DNA, which exists in all cells of the body. Several successive segments of DNA make up a gene, with the human body containing approximately 20,000. Every gene has a different arrangement of DNA segments Read More…
DNA (deoxyribonucleic acid) is the way that living beings store the information that determines how they look and function. Think about DNA as the blueprints, or instructions, for life. All life forms – humans, cats, dogs, trees, and bacteria – all contain DNA. Your DNA is what carries the information Read More…
Purkinje cells are important neuronal cells located in the outer layers of the cerebellum. The cerebellum is part of the brain that is primarily known for controlling sense of balance and movement but can also influence learning, memory, and mood. Purkinje cells receive lots of information from other neurons through Read More…
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