In focus: rare diseases

Check out this round-up of top content for Rare Disease Day 2018!

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Feb 28, 2018
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In honor of Rare Disease Day 2018, we've collated some of our top content highlighting how regenerative medicine therapies can help treat rare diseases. In this focus, watch teasers from an upcoming episode of #TalkingRegMed with special guest Sven Kili, VP and Head of Cell & Gene Therapy Development, GlaxoSmithKline, and find out why rare diseases are so amenable to regenerative medicine and cell therapy. Plus, access news, editorials and reviews as well as free content from Personalized Medicine, free exclusively for RegMedNet members. Join us today!

Keep up to date with everything taking place for Rare Disease Day with #RareDiseaseDay on Twitter.

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Interview: Could gene therapy win the fight against sickle cell disease?

Review: Bringing regenerative medicines to the clinic: the future for regulation and reimbursement

Review: hESC/iPSC-based 3D skin models – customizing therapy of atopic dermatitis

Review: Toward personalized medicine in Bardet–Biedl syndrome (free access exclusively for RegMedNet members)

By default, our videos play muted with captions turned on. Want to hear what our speakers have to say instead of reading it? Click the speaker icon in the bottom left of the video player window.

Interview: 
Building the world’s largest iPSC repository: an interview with Stephen Lin
Commentary: Personalized medicine in rare diseases (free access exclusively for RegMedNet members)
News: Gene therapy shows long-term efficacy in clinical trial for rare cause of blindness
News: Final draft guidelines from NICE recommend gene therapy for rare immune deficiency

Learn more about the ongoing development and delivery of regenerative medicine therapies for rare indications on our cell therapy and clinical trials channels.

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