The use of the gene therapy elivaldogene autotemcel (Skysona), has been approved due to its success in the treatment of patients diagnosed with the neurological disease cerebral adrenoleukodystrophy (CALD). The…
According to a recent report in Healio News, the GLP-1 agonists are medications developed primarily to control blood sugar levels for type two diabetes. About the FLOW Clinical Trial The…
Patient Worthy's award-winning podcast "Wait, How Do You Spell That? A Rare Disease Podcast" is back with a new episode. On this episode of the podcast, we discuss the role…
The Riken Center team, headed by Dr. Takaomi Saido and using mice with Alzheimer’s, discovered that dopamine may lessen the brain’s physical symptoms and improve memory. The study, featured in…
According to a story from Healio, a recent study determined that adults living with Graves' disease who were being treated with antithyroid medications will have an elevated risk of developing…
A protein called Aplp1 is now on the radar for its ability to spread material in the brain that may be responsible for Parkinson’s disease. Another protein, Lag3, interacts with…
According to a story from Healio, analysis of the FDA Adverse Events Reporting System database indicates that tirzepatide has a superior safety profile when compared to GLP-1 inhibitors that have…
This article was written by and shared with permission from Shannon Wieloch, MS. Shannon is a certified genetic counselor and patient advocate for the Aicardi-Goutiere Syndrome Advocacy Association and founder of Stork Genetics.…
Researchers discovered a version of the Cas protein that fits into adeno-associated viruses (AAVs). This virus can be engineered to deliver targeted therapy to cells and is a common approach…
According to a story from Business Wire, the biotechnology company Viridian Therapeutics, Inc., has completed its enrollment goal for its phase 3 clinical trial designated THRIVE-2. This clinical trial will…
According to a story from BioSpace, the biotech company Dianthus Therapeutics recently announced that the US Food and Drug Administration (FDA) has cleared the company's phase 2 clinical trial. This…
It is estimated that over six million people in the U.S. have Alzheimer’s disease plus millions of other people throughout the world. Alzheimer’s is the most common type of dementia.…
Women who undergo allogeneic hematopoietic cell transplantation (alloHCT) face significant treatment-related challenges regarding fertility. However, science seems to be overcoming many treatment-related fertility challenges after alloHCT and successful pregnancies are…
The COURSE Phase IIa clinical trial was conducted to evaluate tezepelumab (marketed as TEZSPIRE) in people living with chronic obstructive pulmonary disease (COPD) that had a wide range of blood…
At Japan’s Fujita University, researchers reported to Inside Precision Medicine that a new risk measurement may add a layer of accuracy in predicting sudden cardiac death. The measure, called estimated…
In some cases, dermatomyositis can be difficult to treat. A variety of treatment options are available, from corticosteroids and immunosuppressants to antimalarials, colchicine, alendronate, and warfarin. But this rare condition…
The long-awaited positive results of Annexon Inc.’s Phase 3 trial investigating ANX005 for Guillain-Barré syndrome (GBS) was presented at the PNS 2024 Annual Meeting. Several leading global GBS experts spoke…
According to a story from PR Newswire, the biotech company Mission Therapeutics was just awarded $5.2 million in funding to support MTX325, the company's investigational Parkinson's disease therapy. Intended to…
According to an article in Science Daily, a drug currently approved to treat the common skin condition eczema may help provide symptom relief in more serious skin diseases as well.…
According to a story from drugs.com, the biopharmaceutical company Palatin Technologies, Inc., which is specializing in the development of therapies that interact with the melanocortin receptor system, recently announced the…
According to a story from Healio, Joe Luvisi, MD, discusses how AI can be utilized to predict a patient's response to anti-VEGF therapy in patients living with diabetic macular edema.…
Individuals who appear to have Long COVID may exhibit a variety of symptoms lasting weeks or even years. The latest estimates are that about 10-20% of people infected with SARS-CoV-2…
A team of scientists from Japan appear to have pioneered a method for reversing synapse damage in an Alzheimer's disease mouse model. Although encouraging, the theory must be tested in…
According to a story from Scleroderma News, findings from the recent Phase 2 EDITA clinical trial suggested that ambrisentan (marketed as Letairis), which is approved to treat pulmonary arterial hypertension…
Long COVID is a phenomenon emerging from the global spread of SARS-CoV-2, a strain of coronavirus causing COVID-19. Seven million people died from COVID-19, though this is considered a serious…
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