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FDA relaxes requirements for manufacturing cell and gene therapies in clinical development Published 14 January 2026 FDA relaxes requirements for manufacturing cell and gene therapies in clinical development Changes to the list of orphan diseases and drugs have come into force in Kazakhstan Published 13 January 2026 Changes to the list of orphan diseases and drugs have come into force in Kazakhstan HAS (France) analyzed the use of real-world clinical practice data in clinical and economic studies of innovative technologies Published 07 January 2026 HAS (France) analyzed the use of real-world clinical practice data in clinical and economic studies of innovative technologies Personalized CRISPR therapy for CPS1 deficiency: Clinical results of the world's first case Published 29 December 2025 Personalized CRISPR therapy for CPS1 deficiency: Clinical results of the world's first case UHC in the CIS countries: key figures and the meaning behind them Published 13 December 2025 UHC in the CIS countries: key figures and the meaning behind them December 12 is Universal Health Coverage Day. Published 12 December 2025 December 12 is Universal Health Coverage Day. Gene-cell therapies in the sights of HTA: An international study has shown that the success of high-tech drugs (HTDs) is determined not only by price Published 10 December 2025 Gene-cell therapies in the sights of HTA: An international study has shown that the success of high-tech drugs (HTDs) is determined not only by price EMA recommends approval of Waskyra, a gene therapy treatment for Wiskott-Aldrich syndrome Published 09 December 2025 EMA recommends approval of Waskyra, a gene therapy treatment for Wiskott-Aldrich syndrome The Organization for Rare Diseases of India (ORDI) is calling for funding for rare diseases based on the Russian "Circle of Good" model. Published 08 December 2025 The Organization for Rare Diseases of India (ORDI) is calling for funding for rare diseases based on the Russian "Circle of Good" model. WHO has released guidelines on best practices for conducting clinical trials: what trials should look like today Published 05 December 2025 WHO has released guidelines on best practices for conducting clinical trials: what trials should look like today FDA approves first gene replacement therapy for SMA available to all ages Published 04 December 2025 FDA approves first gene replacement therapy for SMA available to all ages Uzbekistan: New state program to help children with orphan diseases for 2026–2030 Published 03 December 2025 Uzbekistan: New state program to help children with orphan diseases for 2026–2030 The CIS Orphan Consortium has a new member! Published 12 November 2025 The CIS Orphan Consortium has a new member! Europe strengthens partnerships for rare disease research Published 10 November 2025 Europe strengthens partnerships for rare disease research MCDA in Rare Diseases: A Tool for Accurate and Fair Treatment Evaluation Published 25 October 2025 MCDA in Rare Diseases: A Tool for Accurate and Fair Treatment Evaluation Kazakhstan: Medicines and Innovative Technologies – Experience and Prospects Published 13 October 2025 Kazakhstan: Medicines and Innovative Technologies – Experience and Prospects Orphan diseases and pediatric anesthesiology: the experience of Russia and Uzbekistan Published 13 October 2025 Orphan diseases and pediatric anesthesiology: the experience of Russia and Uzbekistan RWE in Orphan Diseases: International Dialogue and Joint Decisions Published 07 October 2025 RWE in Orphan Diseases: International Dialogue and Joint Decisions Biomedical Collaboration: A Visit to Beijing's Daxing Science and Technology Park Published 06 October 2025 Biomedical Collaboration: A Visit to Beijing's Daxing Science and Technology Park Visit of NAERES representatives to the Beijing Orphan Center Published 01 October 2025 Visit of NAERES representatives to the Beijing Orphan Center A cooperation agreement was signed between the National Medical Chamber of Russia and the Chinese Medical Association. Published 26 September 2025 A cooperation agreement was signed between the National Medical Chamber of Russia and the Chinese Medical Association.

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