Best in Biotech 20 Feb 2025 3 promising biotech approaches to treat Hunter syndrome, a rare genetic disorder Discover three promising approaches being investigated to treat Hunter syndrome, a rare genetic disorder affecting children. February 20, 2025 - 10 minutesmins - By Willow Shah-Neville Share WhatsApp Twitter Linkedin Email
News and Trends 2 Oct 2024 Niemann-Pick disease: FDA approves first two drugs in the space of a week for rare disorder Find out all about the first two FDA drug approvals for the rare genetic disorder Niemann-Pick disease type C. October 2, 2024 - 7 minutesmins - By Willow Shah-Neville Share WhatsApp Twitter Linkedin Email
Podcast 7 Jun 2024Hope for tomorrow: Developing treatments for rare pediatric diseases On this week’s podcast we are discussing novel treatments of rare pediatric diseases with Dan Williams, CEO, executive director of Synaptix Bio. June 7, 2024 Share WhatsApp Twitter Linkedin Email Listen Now Apple Music Spotify More
Podcast 24 May 2024How biotech is tackling myasthenia gravis To talk about the disease, how it is being treated, and what is in the pipeline for future treatments, we had a conversation with Dana Vigier, head of Central Eastern and Northern Europe at Alexion. May 24, 2024 Share WhatsApp Twitter Linkedin Email Listen Now Apple Music Spotify More
In Depth 15 May 2024 Drugs for IgA nephropathy on the rise in the clinic As we observed IgA Nephropathy Awareness Day on May 14th, let us take a look at ongoing clinical trials for drugs that aim to make patient lives better. May 15, 2024 - 9 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email
In Depth 3 May 2024Can emerging new treatments lead the way to cure Duchenne muscular dystrophy? Delve into the new treatment approaches to Duchenne muscular dystrophy, a challenging disease for those suffering from it, and research. May 3, 2024 Share WhatsApp Twitter Linkedin Email
In Depth 14 Mar 2024 Rett syndrome: advances in gene therapy brings hope Explore the latest advancements in treating Rett syndrome, a rare genetic disorder, including groundbreaking gene therapies and drug trials. March 14, 2024 - 8 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email
Podcast 6 Oct 2023Beyond Biotech podcast 66 – treating rare diseases with tRNA Compared to the smallest mRNA molecule, which is 300 nucleotides long, the largest transfer RNAs (tRNA) is less than a third of the size at 76 nucleotides. Scientists at Alltrna are harnessing the unique biology of tRNAs to engineer a single tRNA medicine that could treat thousands of rare diseases that share the same genetic […] October 6, 2023 Share WhatsApp Twitter Linkedin Email
Opinion 20 Sep 2023 The next frontier in genomic technologies for rare diseases By Neil Ward, VP and General Manager at PacBio EMEA It’s been 40 years since the passage of the Orphan Drug Act, which catalyzed the development of hundreds of therapies for rare diseases, with more than half approved in the last decade. More recently in 2022, the U.K. government launched a Rare Diseases Action Plan […] September 20, 2023 - 7 minutesmins - By External Contributor Share WhatsApp Twitter Linkedin Email
News and Trends 14 Jul 2023 Life saved: AI discovers existing drug works for rare disease A patient’s life was saved after researchers at the University of Pennsylvania, Castleman Disease Collaborative Network, Medidata, and Every Cure, used artificial intelligence to use an existing drug to treat idiopathic multicentric Castleman disease (iMCD). Every Cure and its partners are developing medical databases and AI algorithms to unlock new uses for existing medicines across […] July 14, 2023 - 4 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
In Depth 10 Jul 2023 Unleashing the potential: Optimizing mRNA for the discovery of transformative medicines By Dr. Alexander Zehnder, CEO, CureVac The success of mRNA vaccines in COVID-19 propelled the role of this new modality to the forefront of medicine. Not only are new prophylactic mRNA vaccines for various respiratory diseases advancing through clinical development, but mRNA medicines are now being developed in many wide-ranging applications, including cancer and rare […] July 10, 2023 - 8 minutesmins - By External Contributor Share WhatsApp Twitter Linkedin Email
News and Trends 6 Jul 2023 £100M LifeArc program aims to help people living with rare diseases LifeArc, a self-funded, non-profit medical research organization and charity, is launching a new program to invest more than £100 million ($127.2 million) by 2030 to deliver new breakthroughs to improve the lives of people living with a rare disease. The Rare Disease Translational Challenge will bring together researchers in rare disease with LifeArc to get […] July 6, 2023 - 4 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email