SMA Type 1

SMA Type 1, also known as Werdnig-Hoffmann disease, is a severe genetic disorder affecting muscle strength and movement. It occurs because of a deficiency in a protein called survival motor neuron (SMN), which is essential for nerve cells controlling muscles. Babies with SMA Type 1 are usually born with very weak muscles, have difficulty swallowing and breathing, and often show symptoms within the first few months of life. It's the most serious form, and without intervention, it can be life-threatening within the first year. Advances in treatment, like gene therapy, have improved outcomes for some patients.