Neurological disorders are the conditions that affect the most important central and autonomous systems of the human body. They affect important components like the brain, spinal cord, peripheral nerves, cranial nerves, neuromuscular junction, nerve roots, etc.
Things get even more complicated when the patient is a 3-year-old toddler. Henry Saladino suffers from an ultrarare neurological disorder called Alternating Hemiplegia of Childhood or AHC. The disorder can lead to the child to a life-threatening seizure or becoming paralyzed. There is no way to know when it would happen or if there are any chances of survival. The families of the patients suffering from AHC have to be on the constant lookout.
AHC affects one in a million people globally. It does not have a proven treatment as of yet. However, a neurologist and AHC expert believes a treatment can knock down the impact of a genetic mutation that causes dysfunction in Henry's brain. However, the treatment comes at a cost.
Henry's parents, Mary and Anthony Saladino have set up a GoFundMe fundraiser to raise $3 million for a genetic treatment that could be the first-ever treatment for AHC.
The treatment is known as Antisense oligonucleotide (ASO). They are short strings of nucleotides that interfere with RNA processing. They work by helping the body to create the missing 'good protein' or by destroying the 'toxic protein'.
(Input from various media sources)