As we know, many treatments for rare diseases are quite expensive - and this price doesn't even factor in travel, lodging, appointments, and more. To help with this, the government…
Nalla Lawson, who lives in Ocala, Florida, recently shared her story and treatment journey through an article published in UF Health. After suffering a fall back in 2016, she was…
In April 2022, Dr. Adrián Alambiaga successfully defended his doctoral thesis at CEU Cardenal Herrera University. His thesis centered around the exploration of progesterone as a way to stop cell…
According to a story from Medpage Today, a recent study found that a dual immunotherapy approach could be an effective treatment for cervical cancer that had been previously treated, has…
EDIT-301 is an investigational, gene editing medicine developed by Editas Medicine for the treatment of beta thalassemia. As recently reported in the Business Insider, EDIT-301 has just been granted the…
The Retina World Congress, a premier congress to discuss issues in retinal health, recently took place from May 12-15, 2022. During the Congress, various speakers and stakeholders discussed advances…
In Europe, Orphan Drug designation is granted to therapies intended to treat, prevent, or diagnose a rare, life-threatening, or debilitating disease. For the purpose of this status, rare conditions…
According to a story from Globe Newswire, The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recently announced a positive opinion in regards to the experimental…
The new FDA-approved oral formulation of Radicava ORS (edaravone) is more convenient than the intravenous formulation, yet the efficacy and composition remain the same. The oral formulation may also be…
Sophie had a wish, and this month her mother is going to parliament to fulfill that wish. Sophie’s death has brought about the first House of Commons debate on childhood…
Imagine the feeling of working for years with the goal of saving children who have congenital athymia. Then thirty years later the FDA approves a regenerative medicine based on your…
The joy of watching your child grow and meet developmental milestones is not always granted to some parents. Clare’s mother, Allison, described her emotions to CHOP after noticing that Clare…
Acknowledgment: This story is sponsored by Neurocrine Biosciences and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing relevant, vetted,…
For the last 52 years, individuals with Wilson disease have been relying, largely, on the same treatment options. These options, designed to remove copper from the body, include Cuprimine (penicillamine).…
Dr. Omar P. Haqqani, MD FACS is currently the Chief of Vascular and Endovascular Surgery at the Vascular Health Clinics. He has a keen interest in understanding and addressing vascular…
As featured recently in the Cancer Therapy Advisor, a study has been conducted on patients diagnosed with metastatic renal cell carcinoma (mRCC), who also have elevated inflammation, and sarcopenia. The…
Prednisone is one of the more common corticosteroids prescribed to individuals with lupus to help reduce inflammation and decrease symptom burden. Corticosteroids are also prescribed for a number of other…
According to a recent article in Genetic Engineering and Biology News, Washington University and University of Hawaii researchers have uncovered a possible drug development candidate in soil bacterium called…
According to a recent article, a new study is demonstrating how the effects of distal renal tubular acidosis impact a patient’s quality of life. Distal Renal Tubular Acidosis (dRTA) Distal…
When Isabel Blevins was just 16 years old, she caught the flu. While she recovered well at the time, her health seemingly went downhill after this incident. Suddenly, Isabel was…
Necroptosis, a type of regulated cell death, has been implicated in diseases such as inflammatory bowel disease, as well as neurodegenerative diseases. According to a recent article in News Medical…
According to a recent article, the results of a new study show that using recombinant human growth hormone to treat children with idiopathic short stature and growth hormone deficiency aids…
Every year almost half of the fourteen thousand patients who have received bone marrow or stem cell transplants will develop chronic graft-versus-host disease (cGVHD). Currently, there are no options…
According to a recent article, researchers at the WORLDSymposium revealed they are looking into using pentosan polysulfate sodium to treat Maroteaux-Lamy syndrome. MPS VI (Maroteaux-Lamy Syndrome) Maroteaux-Lamy syndrome is…
According to a recent article, the treatment for macular edema associated with uveitis, Xipere, is now available in the United States. Uveitis Uveitis is a term that refers to inflammation…
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