PAEDIATRIC RARE DISEASES
SMDG is highly committed to leverage its novel new class of peptide conjugate nanosystems (PCN)TM and our therapeutic platform technology to design and deliver safe and effective therapies for rare neurological disorders.
"A disease is defined as rare if it has a prevalence of less than 5 per 10,000. However, collectively, rare diseases are surprisingly common, creating a huge health-care and economic burden. There are an estimated 5000–8000 rare diseases, and about 5–8% of the European population is thought to be affected by a rare disease, many of which have neurological manifestations. In the U.S.A, it's estimated that over 25-30 million people live with rare diseases. About 80% of rare diseases have a genetic basis, and onset is often in childhood." The Lancet Neurology
Pediatric Rare Diseases
SMDG is focused on designing safe and efficacious therapies for rare pediatric diseases. One example are neurometabolic disorders which are a group of conditions/disorders causing health problems with both metabolism (that disrupt how the body uses or produces energy from food) and brain function. Children with metabolic disorders may manifest neurological signs and symptoms, as well as developmental delays or known neurological problems that turn out to stem from metabolic disorders. Neurometabolic disorders are complex and may result in uncontrollable epilepsy, abnormal movements or loss of developmental milestones starting at a very early age. These defects may be inherited from unaffected parents (healthy carriers) or may be the result of a new change in a child's genes. Pediatric Neuromuscular disorders affect the nerves, especially those outside the brain and spinal cord (peripheral nerves). They also affect skeletal muscles, such as those in the trunk, arms and legs. These disorders can be stable and unchanging or can worsen over time.