Rare Disease Research Updates

Rare Disease Research Updates

Though rare diseases affect almost 30 million Americans, research challenges and hurdles for therapeutic development persist. The U.S. Orphan Drug Act defines a rare disease as a disorder that affects fewer than 200,000 individuals. These small population sizes make the typical study requirements even more challenging. With pharmaceutical companies less inclined to invest in developing treatments for these disease areas, there is great potential for clinical advancement. Research advancements in one disease may help improve our understanding of other diseases. The FDA has encouraged the study of rare diseases and recently released draft guidance to help advance precision therapies for rare diseases.

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New Developments in Parkinson’s Disease Research

Parkinson's Disease

With Parkinson’s disease (PD) affecting the lives of millions of people, reliable and effective diagnostics and treatments are needed to ease the burden of disease on individuals and society as a whole. With knowledge of the disease growing each year, investment in research has accelerated, offering hope to people living with challenging PD symptoms.

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Clinical Research Updates in Traumatic Brain Injury (TBI)

TBI research updates

Traumatic Brain Injury (TBI), caused by a jolt or blow to the head, or penetrating injury, such as a gunshot wound, is a significant burden for patients and society as a whole. Certain individuals are more prone to experiencing TBIs than others, like those who participate in high-risk sports and activities, such as football, horseback riding, and motorcycling. Others with high-risk careers experience higher rates of TBI, like military servicemembers, first responders, and construction and manufacturing workers.

New research, diagnostics, and treatments are offering hope to people living with the long-term effects of TBI.

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New Advances in Cardiovascular Disease Research and Treatment

New Advances in Cardiovascular Disease Research and Treatment

Heart disease continues to be a leading cause of death in the United States. More Americans die from Cardiovascular diseases each year, including stroke, than cancer and accidents combined, though overall, death rates are declining.

Promising new research into the causes of cardiovascular disease, as well as new therapies and approaches to research and diagnosis, have the potential to revolutionize patient care.

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Clinical Trials and Research to Watch in 2026

Clinical Trials to Watch in 2026

In 2026 there are many exciting clinical trials on the horizon that have the potential to advance research and improve patient care. From new approaches to cancer treatment, to first-in-class treatments for cardiovascular disease and promising drugs for treating long COVID, this year we hope to see study results that indicate progress in personalized medicine and accessibility of care.

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Over 25 drugs being developed in Autistic Disorder pipeline landscape

Autism Spectrum Disorder puzzle illustration

Autistic disorder, also known as Autism Spectrum Disorder (ASD), encompasses a spectrum of neurodevelopmental disabilities. Ultimately, research continues to reveal factors that correlate with ASD risk, but no causal determinations have been made. This leaves much room for discovery with investigators continuing to elucidate new variants conveying genetic risk, or new environmental correlates that require further study.

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North America Emerges as the Hub of Oncology Precision Medicine

Precision Medicine approaches in Oncology

Cancer is recognized as one of the deadliest diseases due to the high prevalence and mortality rates across the globe. To address the limitations of existing treatment options, the concept of precision medicine, often known as personalized medicine, has emerged as a possible solution, which utilizes genomic knowledge to develop gene-targeted therapeutics.

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