Addressing rare disease clinical trial challenges: The power of complex innovative designs
Unlock the power of complex innovative designs to tackle rare disease clinical trial challenges efficiently.
Rare disease
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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.
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 […]
In Depth 7 Sep 2023
Hope for patients with life-threatening muscle disorder after years of clinical trial failures
Duchenne muscular dystrophy is a rare condition that is typically treated with the help of steroids, to manage symptoms, as scientists are yet to close in on a cure. However, the first-ever gene therapy for Duchenne muscular dystrophy is set to open doors for further therapeutic research. As we observe World Duchenne Awareness Day on […]
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 […]
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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 […]
News and Trends 20 Jun 2023
BIO International Convention 2023: The Highlights
BIO International Convention, one of the largest networking events hosted for the biotech industry, has been influential in helping biopharmas discover new opportunities and building and strengthening partnerships within the biotech and pharma community. This year, the event took place from June 5 to 8, in the bustling biotech hotspot of Boston, Massachusetts, and saw […]
In Depth 14 Jun 2023
How are biotechs addressing rare chromosome disorders?
Human beings typically have 23 pairs of chromosomes. But sometimes, in rare cases, an error during cell division can result in cells having extra or missing chromosomes, indicative of chromosomal disorders. Often, prenatal tests are carried out to detect chromosomal abnormalities. Amniocentesis is a diagnostic procedure where the amniotic fluid that protects a fetus during […]
News and Trends 16 May 2023
SiSaf siRNA therapy gets FDA designations to treat autosomal dominant osteopetrosis
SiSaf Ltd, an RNA delivery and therapeutics company, has announced that SIS-101-ADO, its siRNA therapeutic for patients with autosomal dominant osteopetrosis Type 2 (ADO2), has been granted orphan drug designation by the U.S. FDA. Also, due to the serious manifestations of this rare skeletal disorder in children, SIS-101-ADO has been granted rare pediatric disease designation […]
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