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…
Why are rare diseases so difficult to study? Is it just their rarity? Or is it also industry challenges? Unfortunately, industry has a lot to do with it. Clinical Trial…
The Boston Globe recently interviewed the principals in a case brought by the Pelletier family against the Boston Children’s Hospital. The article gives details about a family’s charge of…
Clinical trials and healthcare as a whole are being transformed by new technology. It's essential for clinical trial sponsors to be aware of the new options available to them, and…
Alyla Annac When Alyla Annac was just 19 years old her father passed away from heart failure. He had suffered 3 heart attacks and doctors said there was nothing else…
The Jack McGovern Coats' Disease Foundation, a nonprofit patient organization committed to supporting critical disease research and raising disease awareness, is offering travel grants for recipients to attend the 2020…
Sedation Versus Sleep Sleep and sedation are not synonymous terms, despite the fact that they are often used interchangeably in the hospital setting. The biggest difference? Sleep is always beneficial…
Christine Wright Christine Wright was an active, athletic, healthy mother of three. But in 2007 she started exhibiting strange symptoms that no one could give her an answer to. She…
During the 1990s, researchers had a dream that by 2020, every individual would have access to their own genome. This would aid in their diagnosis and treatment as soon as…
Without understanding the cause of disease it is difficult to understand how to most effectively care for patients. Instead of addressing the underlying cause of disease, physicians are forced to…
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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…
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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…
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…
According to a story from gurufocus.com, the biopharmaceutical company ADMA Biologics, Inc., has earned the 2020 BioNJ Innovator Award. This award was given as acknowledgement of the company's successful development…
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.…
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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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…
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…
Methylmalonic Acidemia Methylmalonic acidemia (MMA) is a rare disease affecting the metabolic system. 60% of all cases are due to a deficiency in MUT, a mitochondrial enzyme, which causes acids to…
Congestive heart failure (CHF) is something that affects about six million Americans, and it currently has no cure. Any treatment that does exist for this condition is supportive and symptomatic.…
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