Fulcrum Therapeutics has recently announced that it is on track to submit an Investigational New Drug application for their medication, FTX-6058. The application will likely be filed in the second…
Ornithine transcarbamylase (OTC) deficiency is a disorder that can cause comas, seizures, and death, making it necessary for those affected to seek treatment. Arcturus Therapeutics aims to give those with…
A three cohort trial of STP705, a treatment for squamous cell carcinoma, has met its primary endpoint. This treatment is administered in a series of injections, offering an alternative to…
Arrowhead Pharmaceuticals has recently submitted an application to the New Zealand Medicines and Medical Devices Safety Authority for the clearance of a clinical trial. They want to test their cystic…
Cabozantinib has recently been studied as a treatment for Ewing sarcoma and osteosarcoma. It demonstrated antitumor activity in both of these cancers, which was discovered in the second phase of…
Crohn's disease is a very rare condition in Taiwan, as only .2 of every 100,000 people are diagnosed with it. Because of this rarity, there is little to no awareness…
Rare, neurologic diseases often do not have viable treatments. Gaucher disease and Parkinson's disease are two examples. In situations like these, medical professionals look to treatments for other conditions, such…
Shannon and Todd Palmatier have three children, two of which have progressive familial intrahepatic cholestasis (PFIC). Their sons' diagnoses have brought the lack of knowledge and advocacy to the attention…
Researchers at Columbia University have discovered a new gene, TBK1, that is linked to the progression of amyotrophic lateral sclerosis (ALS). A lack of understanding of a rare disease hinders…
CAR-T cell therapy has shown to be a viable treatment for mantle cell lymphoma after being studied at the University of Texas MD Anderson Cancer Center. The study showed that…
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Zolgensma is a gene therapy developed by AveXis for the treatment of spinal muscular atrophy. It was approved by the FDA in May of 2019, and was later cleared for…
Phase IIa of the clinical trial of Lu AG06466 has recently concluded. This drug is meant for the treatment of Tourette syndrome, but has not met the primary endpoints in…
APR Applied Pharma Research is a small pharmaceutical company in Switzerland concerned with innovations for rare diseases. They have recently been granted the Orphan Drug designation by the FDA, meaning…
Current treatment for Huntington's disease only relieves symptoms, allowing for the progression of the disease to continue. UniQure, a company that works with gene therapies, aims to change this. They…
Balance issues are often a difficult symptom to detect and track in those with multiple sclerosis. It is important that these problems are addressed by doctors, as they can lead…
Living with a rare disease during this pandemic is scary. There are additional concerns, risks, and worries that must be considered. Those affected by adrenoleukodystrophy have to keep many of…
Research has recently been published in Nature Communications that shows the success of a new gene therapy in the treatment of Huntington's Disease. This therapy, an adeno-associated virus-based gene therapy, was tested…
People affected by rare diseases often find it difficult to find adequate treatment for their conditions. Due to the rarity of their diseases, there are often no doctors who specialize…
There are currently no FDA approved treatments for alopecia areata, a condition that sees the loss of hair all over the body. Eli Lilly and Company, working with Incyte Corporation,…
The first patient has been dosed in the third phase of the VIITAL study, which is intended to evaluate the effectiveness of EB-101. This medication is a gene-corrected cell therapy…
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During the time of a pandemic, it is valuable to look to past pandemics and judge their effects. Eleven years ago, H1N1 swept the world just as COVID-19 is now.…
Lyme disease is typically cured with antibiotics, and those affected never experience symptoms again. But for 20% of those who contract this illness, the antibiotics are ineffective, resulting in lingering…
The COVID-19 pandemic is scary, and it is beginning to cause a panic throughout the world. Through this panic and fear, it is important to remember that progress is always…
LymeDisease.org is one of the most distinguished nonprofits for Lyme disease in the United States. In 2015 they created MyLymeData in the effort to improve treatments for this disease. 10-20%…
Gina Hollenbeck never considered that she could have lung cancer. She did not fall into any categories that would put her at a higher risk of cancer; she didn't smoke,…
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