Rare Pediatric Diseases

RARE PEDIATRIC DISEASES

Our Focus

Rare Pediatric Diseases

We are highly committed to leverage our novel First-in-Class Peptide Therapeutic PlatformsTM and our Peptide small molecules for developing safe and effective therapies for rare neurological dieases.

"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

Rare Pediatric Diseases

SMDG is focused on developing safe and efficacious therapies for rare pediatric diseases.

Rare pediatric neurological motor disorders are a group of complex, often genetic conditions that affect a child’s ability to control movement, coordination, and muscle function. These disorders typically present early in life and can lead to lifelong physical challenges, impacting mobility, communication, and overall development.

While significant progress has been made in genetic and clinical diagnosis, therapeutic options remain very limited. Most conditions still lack disease-modifying treatments, leaving care largely supportive and focused on symptom management. This gap underscores the urgent need for innovative approaches that can translate biological insight into meaningful clinical outcomes for affected children.