Research in Duchenne Muscular Dystrophy (DMD)
Duchenne Muscular Dystrophy (DMD) is a rare genetic disease affecting one in every 3500 boys in which due to lack of dystrophin in muscles affecting muscle dysfunction initially making them wheel chair bound around the age of 15 and early death due to respiratory muscle or cardiac muscle failure. Presently gene therapies are under clinical trials and exon skipping trials thar are showing some progress are available only to a small proportion of the patients as they may target specific gene defects in terms of the portion of the deleted of the exon. As we found the potentials and safety of N-163 strain of Aureobasidium Pullulans produced beta 1,3-1,6 glucans as anti-inflammatory adjuvants in pre-clinical and clinical studies in other illnesses with immune-hyperactivation, two clinical studies in patients with DMD and pre-clinical studies in MDX mice have been undertaken giving a path-breaking outcome.
Importantly this safe, allergen free beta glucan could be consumed by patients of any age even those beyond 18 years in whom gene therapies may not be very beneficial as the satellite muscle stem cells would have got depleted by that age.