Podcast 7 Jun 2024
Hope 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.
Rare disease
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Podcast 24 May 2024
How 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.
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.
In Depth 3 May 2024
Can 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.
Complex innovative designs: tackling rare disease clinical trial challenges
Unlock the power of complex innovative designs to tackle rare disease clinical trial challenges efficiently.
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.
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Podcast 6 Oct 2023
Beyond 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 […]
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 […]
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 […]
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 […]
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 […]
News and Trends 5 Jul 2023
Narcolepsy drug gets Chinese approval
RareStone Group, a rare disease-focused company aiming to establish the first rare disease ecosystem in China, has announced that the Chinese National Medical Products Administration (NMPA) has approved pitolisant (Wakix) for the treatment of excessive daytime sleepiness (EDS) or cataplexy in adult patients with narcolepsy. In China, pitolisant is the first approved innovative drug for […]
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