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,…
Roche has recently been granted priority review by the FDA, who has also accepted their supplemental biologics license application for atezolizumab. Atezolizumab is intended for the treatment of non-small cell…
AVROBIO has recently received the Orphan Drug designation for AVR-RD-04, a gene therapy for the treatment of cystinosis. This treatment takes the stem cells of those affected and modifies them…
An issue with current treatments for Parkinson's disease is that they only provide temporary relief from symptoms. There are no therapies that halt or even slow the progression of the…
Patient confidentiality is often seen as an absolute in the medical world. Many people believe that it should never be broken. Cases do exist that challenge this thought however, where…
Primary sclerosing cholangitis (PSC) is a chronic liver disorder that typically affects males over the age of 30. That is why Di'Jon Thomas was so surprised to be diagnosed with…
Ashley and Chris Numbers are the parents of Alayna and Michael. Soon after their wedding they noticed that Alayna was covered in bruises, which prompted them to take her to…
Sarclisa was recently approved for the treatment of multiple myeloma when taken with pomalidomide and dexamethasone. The FDA indicated that this treatment is for those who have been treated with…
Austin Jackson is a star football player at the University of Southern California. His entire life consists of football, whether that is training for it, watching it, or playing it.…
Prevail Therapeutics has released an update on their Parkinson's disease treatment, PR001. They are currently dosing participants, and enrollment remains open. As of now, the study remains on track. Results…
The European Medicines Agency has recently released their positive opinion of ALUNBRIG, which is a treatment for anaplastic lymphoma kinase+ (ALK+) non-small cell lung cancer (NSCLC). They have recommended this…
Abbey Luffman was a 16-year-old from Owego, New York excited about getting her learner's permit. She was also very generous; she didn't even hesitate to check the box for organ…
Obtaining a diagnosis for a rare disease tends to be very difficult. Doctors do not immediately expect that rare conditions are causing one's symptoms; they blame more common conditions first.…
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