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The mission of the ALS Therapy Development Institute (ALS TDI) is to develop effective therapeutics that stop ALS as soon as possible. Focused on meeting this urgent unmet medical need, ALS TDI executes a robust discovery program, as well as a multi-pronged approach to validate potential therapeutics; including small molecules, protein biologics, gene therapies and cell-based constructs. The Institute's unique, industrial-scale platform allows for the development and testing of dozens of potential therapeutics each year.
Below here some more information on this disease.
For more detailed information, please visit www.als.net
Amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig's disease and outside the US as Motor Neuron Disease (MND) or Charcot's Disease, is a progressive neurodegenerative disease that attacks motor neurons in the brain (upper motor neurons) and spinal cord (lower motor neurons) and affects muscle function.
The motor neurons control the movement of various voluntary muscles including the diaphragm. Associated with the loss of the ability of motor neurons to function in ALS, the various muscles cells waste away (atrophy), resulting in increased muscle weakness. Ultimately, accumulated loss of motor neuron makes it impossible for voluntary control of normal muscle function.
Symptoms of ALS can include twitching and cramping of muscles (called fasciculation), stiffness in muscles (spasticity), increasing loss of motor control in hands and arms and legs, weakness and fatigue, slurred or thick speech and difficulty breathing or swallowing.
In most cases, ALS patients do not typically experience significantly impaired sensory neural functioning, intellectual reasoning, vision or hearing. Eye and bladder muscles, along with sexual function and drive, are not normally affected.
ALS is diagnosed using a variety of tests and examinations, including laboratory tests, muscle and nerve biopsy, spinal tap, X-rays, MRI's and electrodiagnostic evaluation of axon function.
Currently there is only a single therapy approved for treating ALS progression - the drug Rilutek (riluzole) with the accepted, modest benefit estimated to be a three month extension in patient survival.
This significant unmet medical need for this devastating disease is the basis for the research and drug development effort at the nonprofit ALS Therapy Development Institute.
PICTURED: Images from the microscopy suite at ALS TDI.
Read our ALS FAQ for more information about the disease.