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Blood immune cells found to impact cancer survival

The immune systems of cancer patients are highly disrupted, with those who have a higher number of immune cells in their blood having a better survival rate, finds a new study that uses a pioneering technique developed by researchers at UCL and the Francis Crick Institute.

20 Feb 2025

ImmuneLENS method provides new insights into cancer prognosis

18 Feb 2025

AI predicts effective treatment for rare disease using existing medications

After combing through 4,000 existing medications, an artificial intelligence tool helped uncover one that saved the life of a patient with idiopathic multicentric Castleman's disease (iMCD).

5 Feb 2025

Study identifies rare diseases linked to increased COVID-19 mortality risks

An England-wide study of over 58 million people has identified eight rare diseases that carry significantly increased risks for COVID-19-related mortality in fully vaccinated individuals.

30 Jan 2025

Long-read sequencing revolutionizes diagnosis of rare diseases

One in every 10 people worldwide is impacted by a rare genetic disease but about 50% of them remain undiagnosed despite rapid increases in genetic technology and testing.

24 Jan 2025

Research reveals sex differences in microglia response to injury

A collision happens. Someone is hurt, a head injury, a concussion. Just as the first responders arrive to help the person, inside the brain, another "crew" of responders is busy clearing debris and repairing injured tissue.

21 Jan 2025

New insights into rare liver cancer as clinical trial for treatment begins

Like many rare diseases, fibrolamellar hepatocellular carcinoma (FLC) mounts a ferocious attack against an unlucky few-in this case, children, adolescents, and young adults.

17 Jan 2025

Researchers launch startup to revolutionize targeted drug delivery using milk exosomes

Two Nebraska researchers have launched a startup company aimed at bringing to market an innovative method for delivering therapeutics, gene editing tools, plasmids and more to targeted locations in the human body.

17 Jan 2025

Over 500 European patients diagnosed through groundbreaking genetic research

More than 500 European patients with unknown conditions have received a diagnosis through new genetic research.

17 Jan 2025

Long reads can revolutionize genetic diagnosis of rare diseases

The cause of rare diseases is increasingly being detected through genome sequencing, which involves reading the entire human DNA by first breaking it into small pieces-short reads.

13 Jan 2025

Persistent or increasing breast density linked to higher cancer risk

Identifying patterns in breast density changes can improve breast cancer risk prediction, emphasizing the importance of longitudinal assessments in screening.

6 Jan 2025

New Optimer contract to develop bioanalytical tools for RNA therapy

Aptamer Group plc, the developer of novel Optimer® binders to enable innovation in the life sciences industry, today announces the signing of a new development contract with a rare disease biopharmaceutical company.

From Aptamer Group 23 Dec 2024

Two studies show potential for newborn genetic screening

Two studies published today in the American Journal of Human Genetics show the potential for genomic screening in newborns to address high rates of infant hospitalization and mortality in the United States.

9 Dec 2024

SRF awards $230,000 grant to advance research on SYNGAP1-Related Disorders

The SynGAP Research Fund (SRF) has awarded a $230,000 grant to The Jackson Laboratory Rare Disease Translational Center to characterize a mouse model for SYNGAP1-Related Disorders, (SRD), a severe neurodevelopmental disorder marked by seizures, intellectual disabilities, and motor and behavioral challenges.

4 Dec 2024

European leaders urged to develop action plan for rare diseases

The EESC high-level conference held in Budapest urged the new European Commission and the EU Member States to develop a comprehensive policy framework, setting common and measurable objectives to address the rare disease pathway and further increase European cooperation.

2 Dec 2024

Penn engineers cook up a new way for safer, more effective mRNA therapies

Penn Engineers have cooked up a new way to improve mRNA delivery, developing an optimal "recipe" for ionizable lipids - key ingredients in lipid nanoparticles (LNPs), the molecules behind the COVID-19 vaccines and other innovative therapies.

22 Nov 2024

Why predictive retina models could be the future of IND applications

Discover how retina models are revolutionizing rare disease treatment and accelerating drug development.

From Newcells Biotech 22 Nov 2024

ESGCT Conference Takeaways: A specialty laboratory perspective

Discover the latest innovations in cell and gene therapy via Cerba Research's highlights of ESGCT.

From Cerba Research 22 Nov 2024

Study reveals new genetic explanation for dilated cardiomyopathy

A potentially life-changing heart condition, dilated cardiomyopathy, can be caused by the cumulative influence of hundreds or thousands of genes and not just by a single "aberrant" genetic variant, as was previously thought, finds a new study led by researchers at UCL (University College London), Imperial College London and the MRC Laboratory of Medical Sciences.

21 Nov 2024

New clinical trial offers hope for patients with rare immune disease IgG4-RD

A team led by Mass General Brigham researchers demonstrated that inebilizumab reduced the risk of symptoms by 87% in patients with the rare affliction known as immunoglobulin G4–related disease (IgG4-RD)

18 Nov 2024