LI Ruohan, DU Liping, JIE Hengbo, MEI Dan. Exploring the Accessibility by Using the Hydroxycobalamin Preparations of High Concentration in Treating Methylmalonic Acidemia[J]. Journal of Rare Diseases, 2023, 2(3): 406-413. DOI: 10.12376/j.issn.2097-0501.2023.03.011
Citation: LI Ruohan, DU Liping, JIE Hengbo, MEI Dan. Exploring the Accessibility by Using the Hydroxycobalamin Preparations of High Concentration in Treating Methylmalonic Acidemia[J]. Journal of Rare Diseases, 2023, 2(3): 406-413. DOI: 10.12376/j.issn.2097-0501.2023.03.011
shu

Exploring the Accessibility by Using the Hydroxycobalamin Preparations of High Concentration in Treating Methylmalonic Acidemia

  • Department of Pharmacy, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China

Funds: 

National High Level Hospital Clinical Research Funding 2022-PUMCH-B-060

National Natural Science Foundation of China 81974183

More Information
  • Corresponding author:

    MEI Dan, E-mail: meidanpumch@163.com

  • Received Date: December 28, 2022
  • Accepted Date: March 15, 2023
  • Available Online: September 08, 2023
2097-0501/©2023 Editorial Office of Journal of Rare Diseases This is an open access article under the CC BY-NC-ND license (https://creativecommons.org/licenses/by-nc-nd/4.0/)
    Graphical Abstract
    • Abstract

  • Methylmalonic acidemia(MMA) is one of the common diseases in hereditary organic acid metabolism disorder. The disorder is mainly treated by using vitamin B12 which comes in various forms.The use of the methylcobalamin and adenosylcobalamin in China cannot meet the needs of children with MMA. Parenteral use of hydroxocobalamin of high concentration has significant advantages in the treatment of MMA. But, the accessibility to hydroxocobalamin is challenging now.Through the supervision and adjustment of hydroxocobalamin, we explored the strategy of using the drugs. Meanwhile, we are considering the oral use of the hydroxocobalamin as supplement to treatment. Solving the challenges of the hydrococobalamin is helpful to meet the treatment of MMA and to provide a new possibility for the use of medication for rare diseases. The improvement in policy and supervision measures will facilitate the development, involvement, and production of hydroxocobalamin, so that the patients will have a good chance of access to the treatment.

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    • References (52)
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