Mahalakshmi, a 16-year-old, was diagnosed with a rare condition of Lysosomal Storage Disorder (LSD) when she was just four years old. Her condition Mucopolysaccharidosis type 4B is one among many categories of over 45 ultra-rare disorders that does not have a cure.
Unlike her condition, in India around 500 babies are likely to be affected every year by treatable LSDs. Currently, enzyme replacement therapy (ERT) for six of LSDs — Gaucher, Fabry’s disease, Pompe disease, MPS I (Hurler disease), MPS II (Hunter disease) and MPS VI are available.
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