Mary McDirmid, chief financial planning and growth officer, at All Needs Planning, shares ways families with special-needs children can care for their children while also caring for themselves.
We are proud to share a deeply moving feature from ABC7 (NBC7) San Diego highlighting the journey of 5-year-old Kiaan Patil and his family’s brave battle against GM1 Gangliosidosis.
As the year comes to an end, we remain focused on creating connection by keeping our rare disease community informed of advocacy opportunities, research news and community updates. Join us in learning more about our efforts to push for a cure for GM1 gangliosidosis.
For families navigating a GM1 gangliosidosis diagnosis, the research landscape can be confusing due to rapid changes in the biotech industry. Several commercial sponsors have faced bankruptcy or shelved programs, shifting the focus back to academic-led studies and preparing for new studies.
GM1 gangliosidosis is a rare genetic disorder that deeply affects families. When parents hear this diagnosis, the first question that comes to mind is often: “What is the life expectancy?”
This guide breaks everything down in simple terms what GM1 gangliosidosis is, how it affects life expectancy, and what families can realistically expect today.
When you’re a parent, you notice everything: every smile, every tiny movement, every missed milestone. Sometimes, those little details are just part of growing up. Other times, they’re quiet warning bells asking to be heard. GM1 gangliosidosis is one of those rare genetic disorders where early awareness can make a meaningful difference for families.
GM1 is classified based on the age of onset, which serves as a primary indicator of how the disease will progress.
GM1 gangliosidosis is one of those diagnoses that can turn a family’s world upside down in a single appointment. One moment you’re searching for answers to unexplained symptoms, and the next, you’re learning about a fatal rare genetic condition most people have never even heard of.
Currently, there is no approved treatment or cure that stops or reverses the disease. However, we are in a “pivotal era” of research. Today, treatment means a dual approach: Expert Symptom Management and Investigational Research.