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 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 gurufocus.com, the biopharmaceutical company Abeona Therapeutics, Inc., recently announced the presentation of positive interim findings from two different phase 1/2 clinical trials. These trials were…
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…
The publication Being Patient tracks the latest news about Alzheimer’s disease. This month it published an article about a new Alzheimer’s clinical trial that is being conducted with the…
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…
The validity of Morgellons disease, which has been associated with Lyme disease, has been widely debated for years. Those affected by it swear that it is real, that fibers grow…
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…
As originally reported in BioPortfolio, a novel DNA therapy for patients with HPV 6 who experience recurrent respiratory papillomatosis (RRP) has concluded its first clinical trial with promising results. As…
Janet Woodcock, Director of the FDA’s Center for Drug Evaluation and Research (CDER) recently gave an interview to the publication FDA Voices about the agency’s approval in 2019 of a…
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…
AskBio Asklepios BioPharmaceuticals (AskBio) was first founded in 2001. It is based out of North Carolina. This company is dedicated to the research of gene therapies as potential therapeutic options…
One of our partner organizations, the MDS Foundation, is hosting another free MDS Patient & Family Forum in Chapel Hill, NC. When: September 26th Where: The Siena Hotel 1505 East Franklin Street…
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…
I recently had the pleasure of being interviewed by Sunshine Mugrabi about how I transformed my life over the past 20 years in the face of immense pain and suffering…
According to a story from Financial Buzz, the gene therapy company AVROBIO recently presented updated data on February 10th, 2020 at the 16th Annual WORLDSymposium being held in Orlando, FL. These updates…
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…
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