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Navigating the Landscape of Rare Diseases: Current Trends and Future Perspectives Neeta Ratanghayra
September 29, 2024
Rising Investment and Market Dynamics, Future Perspectives in the Orphan Drug Market and The Scenario in China.
Rare Disease Drug 101 - What are the Benefits to Apply for and Receive Orphan Drug Designation? Lin Zhang
June 29, 2021
There are many common diseases that affect millions of people across the world. On the other hand, there are so-called rare conditions that affect a small number of people.
China increases accessibility to rare disease treatment NMPA/Xinhua
December 24, 2021
Two-and-half-year-old Xuanxuan survived a rare kidney disease in early December, thanks to fast-track approval of the transplant.
Stealth BioTherapeutics Joins the Rare Disease Company Coalition firstwordpharma
July 08, 2021
Stealth today announced that it has joined the Rare Disease Company Coalition, an alliance of life science companies committed to discovering, developing, and delivering rare disease treatments for the patients they collectively serve.
Low patient accrual rates cause termination of over 25 per cent rare disease trials: GlobalData expresspharma
March 11, 2021
Difficulties with patient recruitment for rare disease clinical trials could be mitigated by the use of virtual trials.
DHSC publishes ‘UK Rare Disease Framework’ pharmatimes
January 13, 2021
The UK’s Department of Health and Social Care (DHSC) has published its ‘UK Rare Disease Framework’, which will aim to continuously improve the lives of people living with rare diseases.
University of Oxford links up with Evox Therapeutics pharmatimes
November 19, 2020
The University of Oxford’s Harrington Rare Disease Centre has entered a strategic collaboration agreement with Evox Therapeutics focused on rare diseases within the Department of Paediatrics.
FDA Awards Grants for Rare Diseases Clinical Trials americanpharmaceuticalreview
October 15, 2020
The U.S. Food and Drug Administration (FDA) has awarded six new clinical trial research grants to principal investigators from academia and industry totaling over $16 million over the next four years.
Cycle Pharmaceuticals secures $25m debt financing pharmaceutical-business-review
September 23, 2020
Cycle Pharmaceuticals, a privately-owned, global, patient-dedicated biotechnology company focused on developing and delivering medicines for rare diseases, announced that it has executed a definitive agreement for a $25 million credit facility with ...
Licensing agreement deals decline in rare diseases space, says report europeanpharmaceuticalreview
August 12, 2020
New research has shown that complex collaborations and partnerships are increasing, while licensing agreements are decreasing in the rare disease space.

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