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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ALS News Today recently announced the introduction of a Phase 3 clinical trial for oral edaravone, a drug that has previously been approved for slowing the decline of physical…
By Danielle Bradshaw from In The Cloud Copy 13-year-old Cam Howard, a young Pittsburgh resident, recently did an interview with the Pittsburgh KDKA News’ Kristine Sorensen where he opened up…
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According to a recent article in ALS News Today, the FDA’s Orphan Status designation has been granted to NeuroSense Therapeutics for its drug, PrimeC, to treat ALS. PrimeC received the designation…
Lennox-Gastaut syndrome (LGS) is a form of epilepsy. Most patients with this condition are resistant to current treatments and experience seizures throughout the duration of their life. They may face…
A recent study led by Morten Aagaard Nielsen and Tue Wenzel Kragstrup, who both work at Aarhus University in Denmark, hopes to help physicians determine the best personalized treatment for…
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…
Hypotonia Ataxia, and Delayed Development Syndrome Hypotonia, Ataxia, and Delayed Development Syndrome (HADDS) is caused by mutations in the EBF3 gene which is found on chromosome 10q26. Its discovery just…
According to a story from Targeted Oncology, findings from a recent phase 1 trial have discovered a new potential treatment for myelofibrosis. The study combined two different drugs: panobinostat (marketed…
Angiosarcoma Angiosarcoma is a rare condition caused by a tumor within the endothelial cells. These cells line the blood vessels in the body, and therefore the tumors can occur practically…
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