The FDA approved the first treatment for penicillamine-tolerant patients with Wilson's disease. Wilson's disease is a rare genetic disorder in which copper transport disorders occur. The first Wilson's disease treatment, Penicillamine, was approved 50 years ago. It is known that about one-third of Wilson's disease patients eventually develop penicillamine intolerance. Therefore, Orphalan's treatment is expected to be an effective treatment with long-term use.
FDA가 Penicillamine 내성이 있는 Wilson병 환자에 대한 치료제를 최초로 승인 했습니다. Wilson병은 구리 수송 장애가 발생하는 희소 유전 질환입니다. 최초의 Wilson’s Diseae 치료제 Penicillamine은 승인된 지 50년이 지났습니다. Wilson병 환자의 1/3은 결국 penicillamine 불내증이 된다고 알려져 있습니다. 따라서 Orphalan의 치료제는 장기간 사용할 수 있는 효과적인 치료제로 기대됩니다. Original Article: MedPage Today.First Drug Approved for Wilson's Disease in Decades
Genesis Therapeutics has signed a new drug development contract with Eli Lilly. Genesis will discover new drug candidates through AI and machine learning technology, and Eli Lilly will take the rights to the candidates. Genesis signed a new drug development contract with another large pharmaceutical company following the deal with Gennectech two years ago.
A study was published in Nature Biomedical Engineering that a group of rare diseases called ciliopathies can be studied using cilia-free human pluripotent stem cells. In this study, researchers removed cilia from normal human pluripotent stem cells and confirmed that the clia-free stem cells produced showed ciliopathy-like symptoms. By comparing normal stem cells with stem cells without cilia, the researchers plan to investigate the molecular biological mechanisms of disease.
Cilia-free 인간 만능 줄기세포를 이용해서 ciliopathies라고 불리는 희소 질환군을 연구할 수 있다는 연구가 Nature Biomedical Engineering에서 발표되었습니다. 이 연구에서 연구자들은 normal 인간 만능 줄기세포에서 cilia를 제거했으며, 이렇게 만들어진 clia-free 줄기세포가 cliopathy-like 증상을 보이는 것을 확인했습니다. 정상 세포와 섬모가 없는 세포를 비교해 질병 발생 기전을 분자생물학적으로 밝혀낼 예정입니다. Original Article: Drug Target Review.Cilia-free stem cells could open a new way to study rare diseases
This is an interview article about why it is challenging to develop a treatment for rare diseases like Huntington's disease. It has been 30 years since the genetic cause of Huntington's disease was identified, but no treatment has been developed to this day. Since the Huntington gene (HTT) functions in complex and important ways in the body, there is a difficulty in maintaining the function of Wild Type HTT while lowering the function of the pathogenic HTT. Dr. Christian Landles of the UCL Queen Square Institute of Neurology introduces Huntington's disease treatment development status and treatment development strategies.