Strimvelis, (autologous CD34+ cells transduced to express ADA) the first ex vivo stem cell gene therapy has been approved to treat patients with ADA-SCID (severe combined immunodeficiency due to adenosine deaminase deficiency). Children born with ADA-SCID do not have a heathy, fully functioning immune system and are unable to successfully combat everyday infections.
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Following a successful presentation at the Cell Therapy Manufacturing and Gene Therapy Congress in Brussels, Onyx has again had a strong presence at the prestigious World Orphan Drugs Congress in Washington. This emphasises Onyx strong and continuing commitment to the areas of orphan drugs, rare and ultra-rare diseases and regenerative medicine including cell and gene therapies.
read more >>We are excited to be speaking at the Cell Therapy Manufacturing & Gene Therapy Congress about the importance of Medical Communications for Gene Therapy. Find out more on KNect365 https://knect365bioprocessing.com/2016/01/29/prime-medical-group-value-medical-communications/
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