When you think about genetic testing, what first comes to mind? Is it pancreatic cancer? While this might not be the first thing you consider, a new study suggests that…
This past month, the Ice Bucket Challenge was brought back into the spotlight when co-founder of the ALS Ice Bucket Challenge, Pat Quinn, unfortunately passed away. As the ALS Association…
In a recent press release, the SynGAP Research Fund (SRF) shared that the Heller Laboratory of Neuroepigenetics would receive a $130,000 research grant. The fund, which will be granted over…
According to Charcot-Marie-Tooth News, biotechnology company AcuraStem recently identified 37 potentially therapeutic compounds that could be used to treat patients with Charcot-Marie-Tooth disease type 2A (CMT2A). Currently, CMT2A is the…
In mid-November, 2020, Rocket Pharmaceuticals announced that the company received a $3.7M grant from the California Institute for Regenerative Medicine to study potential treatments for malignant infantile osteopetrosis. In…
From November 5 through 9th, the American College of Rheumatology (ACR) held their ACR Convergence 2020. Though the annual meeting took place online, it was still rife with insights into…
After announcing positive results from a Phase 2b ADVISE trial, Arena Pharmaceuticals, Inc. ("Arena") hopes to soon initiate a Phase 3 clinical trial to evaluate etrasimod as a treatment…
Recently, biopharmaceutical company Everest Medicines ("Everest") announced positive topline data from the Phase 3 NefIgArd trial, which was run by its licensing partner Calliditas Therapeutics AB ("Calliditas"). Together, the…
According to Neurology Today, researchers have discovered a new way to use artificial intelligence (AI) to enhance medical knowledge. Through the DystoniaNet platform, which uses deep learning AI, researchers targeted…
In a recent study published in Clinical and Experimental Immunology, researchers determined that patients with high ANCA antibody levels are at an increased risk of having ANCA-associated vasculitis (AAV) relapses. According…
Many people within the rare disease community share a common experience: feeling isolated or misunderstood because of your condition. In some cases, such as conditions causing chronic pain or fatigue,…
At the very core of genetic diseases are - well, our genes. However, each year, an estimated 400,000 infants are born with de novo mutations, or new and spontaneous gene mutations not…
Recently, during the virtual 28th United European Gastroenterology Week Congress, the Janssen Pharmaceutical Companies of Johnson & Johnson announced 5-year results from a Phase 3 extension study on STELARA…
A biological marker ("biomarker") is a measurable trait or characteristic that can tell researchers something about your body or health. For example, body mass index (BMI) is a common biomarker…
For many years, researchers linked obesity to a number of other health conditions. Now, Taiwanese researchers identify a new cause for concern: worsening health outcomes for patients with ankylosing…
Did you know that testing protein levels in our bodies can tell researchers about our health? In some cases, it can even give insight into disease progression and development.…
I'm sure you have heard about Zika virus before, but what about Chikungunya? Both are rare mosquito-borne diseases that can cause severe neurological problems. But now, researchers also believe…
Researchers have known, for a while, that a variety of gene mutations contribute to different subtypes of Charcot-Marie-Tooth disease. The specific condition is dependent on which gene is mutated. But…
This year, the American Society of Retina Specialists (ASRS) held their annual meeting online. Many leaders in the field, such as Dr. Bobeck Modjtahedi, MD, attended to present new…
What do you call fossils that just lie there? Lazy bones! But for the Centrosaurus apertus, its bones are talking to us from beyond the grave, making it less lazy and…
When Zika virus appeared in the news in 2015 and 2016, one of the biggest warnings was for pregnant women: be careful, as this can cause birth defects. Most…
People with genetic diseases or disorders may experience differences on a physiological level. For example, shares Medical XPress, fragile X syndrome (FXS) results in less primary cilia. Since primary cilia…
Currently, over 200 different mutations are linked to the development of best vitelliform macular degeneration (BVMD, or Best disease). But while certain treatments may treat certain mutations, there has…
What causes developmental disorders? How can the human genome play a role in diagnosing and treating these conditions? Well, a team of scientists from the Salk Institute recently set…
As of right now, there are no treatments for Alzheimer's disease, a progressive neurodegenerative disorder. However, that may soon change. A study led by UCLA researchers identified potential drug…
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