藥物開發和治療為罕見疾病帶來新希望

TAIPEI, TAIWAN, Sep. 30, 2021 - In the past, rare diseases tended to be very rare genetic disorders that are difficult to diagnose. However, more than 350 million people in the world are suffering from these rare diseases. As genetic sequencing technology progresses, genomics study helps to accelerate the discovery for rare disease diagnosis. Leading genomics in revolutionizing the approach of new diagnosis and treatment for rare disease. Many thrilling collaboration news recently showed that industry leaders in pharmaceutical companies, hospitals and national institutes start to focus more on drug development and treatment of rare disorders.

Diagnosis and Treatment for Rare Disease

The diagnosis is so important for the genetic disease patients and their families. The use of genetic testing is not only effective for diagnosis but also helps save the cost, through shorter hospital stays, fewer invasive procedures and targeted personalized care. These advantages would encourage the patients, their families, and the attending physicians.

While genomics have made great progress in diagnosing rare genetic diseases, the fact is that only around 10% of childhood genetic diseases currently have effective treatments. As the drug development usually takes years or even decades of research and trials, the treatment would be highly expensive. The rare disease drug is also called “Orphan Drugs”, because the pharmaceutical industry has little interest under normal market conditions in developing and marketing drugs intended for only a small number of patients suffering from very rare conditions.

Taketa is Digging into Pharmacogenomics for Rare Disease

In July 2021, the world biopharmaceutical leader Takeda partnered with the California based institute - Rady Children’s Institute for Genomic Medicine to develop treatments for rare genetic diseases. Taketa also announced the collaboration with Taiwan National Health Research Institute for rare genetic disease – Fabry disease drug development in 2020.

Building the foundation for genomics in precision medicine

Through collecting the huge amount of patient’s genetics datasets, researchers can extract a lot of hereditary information for specific disease cohorts. It leads drug development into a new era. Being a leading genomics bio-IT company in Taiwan, we are very pleased to know that more and more drug R&D are built upon pharmacogenomics and genetic study. We hope the world with personalized treatments and precision medicine will come very soon for everyone in medical needs.

References:

[1] Rady Children’s and Takeda Partner to Develop Treatments for Rare Genetic Diseases

[2] Rady Children’s Helps California’s Project Baby Bear Improve Outcomes, Save $2.5M

[3] About orphan drugs

[4]National Health Research Institutes and Millennium Pharmaceuticals, Inc., a wholly-owned subsidiary of Takeda Pharmaceuticals Company Limited, join forces in the area of genome research to develop rare disease drugs

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