Cell-in-a-Box, the name of one of Biotech’s new products is just as intriguing as the drug itself. A recent article in NewsFile characterizes the product as having the potential…
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…
As originally reported in Pfizer, the European Commission has just approved the first treatment option ever available to treat transthyretin amyloidosis cardiomyopathy (ATTR-CM). The medication, VYNDAQEL, taken orally once daily,…
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…
For years scientists have tried to understand how and why tau, well known to researchers for its implication in Alzheimer’s disease, changes from its normal form to a harmful…
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…
February 16, 2020 The doctor wasn't listening to me. He didn't care that I was suffering. “I’m so sorry, I thought we were doing the right thing,” my dad said,…
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…
By Natalie Homan from In The Cloud Copy Theo Thomas of Secret Harbour, Australia, may be a shy, soft-spoken 8-year-old, but he loves racing his motorcycle in his town’s Lightweight…
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…
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