Rare diseases require national level cooperation to be properly addressed given (i) varying types and onset patterns resulting in difficulties for accurate diagnosis (ii) lack of experienced medical professionals serving as a hurdle to conduct advanced research and (iii) high treatment costs throughout the patient’s life.

Novel Pharma is pursuing therapies designed to pass through the blood-brain barrier to improve the quality of lives of people suffering from degeneration of the central nervous system due to genetic causes.

Novel Pharma started joint research with the Association for Research of MPS and Rare Diseases to develop rare disease therapeutics with the sole purpose of solving social and daily difficulties of patients and their families suffering from rare diseases.

Association for Research of MPS and Rare Diseases (ARMRD)

ARMRD is the largest association for research of mucopolysaccharidosis (MPS) and other rare diseases in Korea, playing a pivotal role in diagnosis and research of rare diseases in Asia through active exchanges among domestic and overseas medical staff. In addition to research on the natural progress of various rare diseases, including MPS, ARMRD has continued to collect disease-related information and research treatments through its worldwide clinical networks. Led by Dr. Dong-Kyu Jin, president of the Association, ARMRD has succeeded in developing Hunterase ICV, the world’s 1st treatment for severe-type Hunter syndrome and proven its potential.

  • Research for screening of patients with rare inborn errors of metabolism
  • Publishing guidelines that reflect the characteristics and circumstances of patients with rare diseases
  • Building patient registration systems for rare diseases
  • Hosting symposiums on rare diseases and supporting patient networks
  • Research for natural progress and treatments of rare diseases

Meet Our Leadership

Our R&D is led by a proven new drug development team with experience in developing Hunterase, the first commercially successful new biologic in Korea.


Our Research in Rare Neurodegenerative Diseases

Novel Pharma is expanding its rare disease therapeutics pipeline based on the experience in the entire cycle of developing new drugs for rare diseases, secured through the success of Hunterase.