In an article in Medical Xpress, Japanese doctors have performed a “first” by transplanting liver cells (made with embryonic stem cells) into a newborn. The six-day-old infant was diagnosed with…
As reported in Guru Focus, biotech company Protagonist Therapeutics has announced progression in their Phase 2 study on PTG-300 as treatment for patients with polycythemia vera, a rare cancer. Dr.…
According to a story from prnewswire.com, the Chiesi Global Rare Diseases and the biopharmaceutical company ProTalix Biotherapeutics have recently announced that they have submitted a Biologics License Application (BLA) for…
Passage Bio has been granted the Rare Pediatric Disease designation for their GM1 gangliosidosis treatment, PBGM01. This designation means that Passage will receive a priority review voucher, and it is…
The medical realm is changing - but how does that impact patient care and clinical expectations? The National Hemophilia Foundation discusses how clinical expectations for patients with hemophilia don't necessarily…
Every day, medical researchers look to learn more about rare diseases, symptoms, and treatment. Recently, the Muscular Dystrophy Association (MDA) connected with Dr. Priya Sunil Kishnani, MD, MBBS to discuss what…
By Danielle Bradshaw from In The Cloud Copy It has been shown in a recently published trial that satralizumab could potentially reduce the risk of neuromyelitis optica spectrum disorder (NMOSD)…
By Jodee Redmond from In The Cloud Copy Dr. Kristina Simonyan has been studying patients with laryngeal dystonia. Through the scope of her work, she has heard numerous anecdotal accounts…
As a child, my favorite fairy tale was about a princess who is locked away in a tower and guarded by a terrible dragon. Finally, a prince comes along, slays…
A recent article in US News and World Report gives an account of the first patient to have reprogrammed cells successfully implanted in his brain. The patient’s doctors reported that…
Immunotherapies are in the forefront for treating autoimmune diseases and cancer. Northwestern University and the University of California San Francisco conducted a study using genes that have the potential…
According to a story from Guru Focus, the biopharmaceutical company Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc. recently announced that the Ministry of Health of Spain has cleared…
Protalix BioTherapeutics has recently announced the results from the third phase of the BRIDGE trial, which evaluated pegunigalsidase alfa for the treatment of Fabry disease. As these results were positive,…
Cytokine storms have been prominent in the news lately due to their role in COVID-19, but researchers have been studying their connection to Castleman disease for a long time. In…
A recent article in Biospace reported an 83% survival rate for COVID-19 patients after two infusions of the experimental drug Ryoncil, developed by Mesoblast Ltd. The patients who were…
The FDA has recently granted GTX-102, a treatment for Angelman syndrome, the Fast Track designation. This designation means that the review process is expedited. It is meant specifically for drugs…
Axcella, a biotechnology company based out of Cambridge, Massachusetts, has released the results of AXA1125-003. According to Biotech 365, this study showed positive results for those with non-alcoholic fatty liver…
by Danielle Bradshaw from In The Cloud Copy A year-long study has shown that Tracleer greatly reduces the skin-related symptoms of scleroderma. The results also showed that although it helped…
A total of one hundred vaccines are being developed by researchers, drug manufacturers, and governments worldwide in an effort to stem the tide of the COVID-19 virus. Reuters Newswire reports…
According to a story from Xconomy, the drug company PTC Therapeutics has come to an agreement that will allow it to acquire Censa Pharmaceuticals and its lead product candidate. This…
According to a recent article in BioMed Central, an international task force formed in 2018 created guidelines for the treatment of juvenile idiopathic arthritis (JIA). Treatment targets were identified…
According to a recent announcement by AstraZeneca, the U.S. FDA granted its approval for Koselugo (selumetinib). This is the first drug to be approved for the treatment of children…
Recently, Pfizer's Dr. Ian Winburn sat down with Europabio for an interview in honor of World Hemophilia Day, which just occurred on April 17. In his discussion, Dr. Winburn…
Phase III of the CheckMate -9ER trial has reached its primary endpoint of progression free survival in those with advanced or metastatic renal cell carcinoma. This study was evaluating CABOMETYX…
by Danielle Bradshaw from In The Cloud Copy Hereditary angioedema (HAE) is a rare condition in which a person has swelling beneath their skin (which can occur in any part…
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