Lalith is a father of two children with Sanfilippo syndrome, both enrolled at Tamahar. Below is one of his blog posts, where he shares his family’s story and his hopes for the future of research.
Sanfilippo syndrome (MPS III) has become a way of life for me and my wife. As parents of two children with this deadly disease, our time is spent in a mixture of living life to it’s fullest and at the same time being very anxious for any signs of regression.
We have a daughter who will be 10 years old in a couple of weeks and a son who is seven years old. My daughter’s birth and the subsequent years were sheer bliss. Things only became concerning when we noticed that her verbal capabilities did not pick up. Visits to various doctors yielded a common answer and it was that she had ADHD (attention deficit hyperactivity disorder). We were assured that a few different therapies would mean she would be fine. But that was not to be. At this point in time our son was born. We were completely ignorant of what my daughter had and had no way to know that the same condition would also affect our son.
Even being in a city like Bangalore, India, that prides itself in being technically advanced and providing access to the best healthcare, it still took a few years for us to arrive at a diagnosis. My daughter was six years old when the Sanfilippo syndrome diagnosis was confirmed. Life suddenly looked different. I can still picture us literally praying for a miracle while the rest of the world seemed to be nonchalantly proceeding ahead as they were blessed with a normal life. The fact that there is no medicine to cure them was made even worse when we were told that their quality of life would deteriorate. There was no choice but to push ourselves out of this attitude of feeling victimized and to instead look towards hope.
I started reaching out to organisations working for kids and caregivers affected by rare diseases. The Organisation for Rare Diseases India (ORDI) is playing a big role here. Knowing that there is an organisation fighting for the cause is reassuring. Similarly there is a special school called Tamahar where my kids were admitted and they have been a source of immense support.
Encouraging news from research companies like Abeona Therapeutics and Lysogene has given us hope. But when we see our kids crying or laughing for a reason we cannot figure out, see them running and suddenly fall down, or see them looking up to us with a pale face when they are hungry as they cannot verbally convey it to us; our hearts sink. We just cannot wait for a treatment that can cure my children and change the lives of our entire family.
I look to the Sanfilippo research community to enable me in getting access to the treatment in form of gene therapy for my kids. As a never-say-die parent, I make myself completely available to anyone who can help me in this endeavor and look towards all of you for your support.