One of the less well known forms of primary immunodeficiency is called activated PI3K delta syndrome (APDS). It is a rare disease that has only recently been identified by scientists.…
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The gene therapy Zolgensma recently won approval last summer in 2019 to be used for infants who have spinal muscular atrophy. The company AveXis, the makers of the Zolgensma gene…
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…
By Danielle Bradshaw from In The Cloud Copy Neurofibromatosis type 1 (NF1)-related plexiform neurofibromas (pNFs) is a condition that many children struggle to manage due to a lack of proper…
According to a press release from Epizyme, Priority Review has been granted for tazemetostat (Tazverik), a drug used to treat relapsed or refractory follicular lymphoma (FL) for patients whose symptoms…
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According to a recent article in The Pharma Letter, the European Medicines Agency (EMA) granted PRIME (PRIority MEdicines) designation to Viralym-M based on positive results from a Phase 2 proof-of-concept…
By Jodee Redmond from In The Cloud Copy A recent study suggests that certain antibodies could be linked to specific human leukocyte antigen (HLA) genes and environmental components in scleroderma.…
By Danielle Bradshaw from In The Cloud Copy A Tufts University science team was able to identify a means to possibly reverse Friedreich’s ataxia. Friedreich’s ataxia (also known as…
By Gracie Van Brunt Hi! My name is Gracie Van Brunt, I am 25 years old and I have a rare disease. At the age of 2, I was…
By Natalie Homan from In the Cloud Copy Most people have heard of type 1 and type 2 diabetes. Many do not know, however, that there is an “intermediate” form…
According to a story from Financial Buzz, the ophthalmology company Acucela Inc. has recently announced an update on the progress of its phase 3 clinical trial. This clinical trial is…
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…
According to a story from stcatherinesstandard.ca, former firefighter Tom Salisbury was 54 years old when he was first diagnosed with a form of vasculitis, a type of rare autoimmune disease.…
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…
According to a story from Healio, Cushing's disease, a cause of Cushing's syndrome, can still be a deadly disease if it remains untreated. However, recent developments in the treatment of…
By Rachel Whetstone from In The Cloud Copy Many patients with myelodysplastic syndromes (MDS) are elderly when they’re diagnosed with the disease. Although stem cell transplants are often thought to…
By Jodee Redmond from In The Cloud Copy Living with a chronic disease is a very stressful situation. It affects a person physically, mentally, emotionally, and spiritually. Chronic illnesses often…
According to a story from Newsfile, the biotechnology company PharmaCyte is beginning to turn heads with the potential of its proprietary Cell-in-a-Box technology that has the potential to greatly improve…
According to a story from Benzinga, the biotherapeutics company CSL Behring has recently announced that its medical product PRIVIGEN ® has earned Orphan Drug designation from the US Food and…
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The ASCO Post recently reported findings of high response rates and improved survival from the combination of platinum-based cisplatin and gemcitabine (chemotherapy) in BRCA/PALB2 mutation positive pancreatic ductal adenocarcinoma. The goal…
As originally reported in CNN, a duck billed dinosaur of a time long before humanity has been identified to have suffered from the same rare disease found in children today.…
Happy Almost-Friday! This week, we're highlighting two organizations: one helps patients seeking gene therapy learn about the treatments, and the other helps families affected by a newly-recognized condition. After that,…
One of the wealthy regulars on the TV show Shark Tank made his millions by starting a home-based business selling tee shirts. An article in Bridgeport’s ctPost publication chronicles…
By Jodee Redmond from In The Cloud Copy Four United States senators have introduced a new law that, if passed, will assist children living with rare disease to receive tests…
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