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.…
Lyme disease is the most common vector-borne disease, and it has become more prominent due to climate change and urban developments spreading further into wooded areas. Treatment is typically effective…
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ACADIA Pharmaceuticals, working with Neuren Pharmaceuticals, has recently been granted the Rare Pediatric Disease designation from the FDA for their therapy Trofinetide. This drug is intended for the treatment of…
Rare diseases often go undiagnosed, which leads to a lack of treatment and support for those affected by them. People with acromegaly, a rare hormonal disorder, have called for change.…
Mantle cell lymphoma is a cancer that often responds well to initial treatment. Because of this, doctors often recommend intense treatment soon after diagnosis. While it is often successful, sometimes…
Lucy Lintott is the youngest woman in Scotland to be diagnosed with motor neurone disease (MND). As it causes progressive weakness, Lucy did not think it was possible for her…
The Carroll family recently welcomed their daughter Renesmee into the world. They immediately checked for symptoms of Batten disease, a rare, fatal nervous system disorder, as two of Renesmee's siblings…
CSL Behring has recently received the Orphan Drug designation for Privigen for the treatment of systemic scleroderma. Privigen has also been granted the Fast Track designation. This therapy has been…
Teva Pharmaceuticals recently finished two clinical trials of their Tourette syndrome treatment, deutetrabenazine. These two studies, titled ARTISTS 1 and ARTISTS 2, did not meet their primary endpoints, as they…
Amyloidosis is a rare disease that has gone without awareness for a long time. This lack of awareness led to slow developments in treatment, issues with obtaining a diagnosis, and…
Poly ADP-ribose polymerase (PARP) inhibitors have played an increasing role in the treatment of ovarian cancer. They are being very heavily studied in clinical trials, both for the treatment of…
The validity of Morgellons disease, which has been associated with Lyme disease, has been widely debated for years. Those affected by it swear that it is real, that fibers grow…
Roche has recently released the results of the latest phases of their study of gantenerumab, which is a treatment of a rare form of Alzheimer's disease. This form, which is…
Prevail Therapeutics has recently been granted the Orphan Drug designation by the FDA for their gene therapy PR001, which is intended to treat Gaucher disease. This treatment has also received…
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