On the field at the University of Tennessee, defensive back (DB) Trevon Flowers was known for his intense gameplay, strong athletic ability, and defensive nature. He appeared in 46…
According to an a press release published on Business Wire, Arrowhead Pharmaceuticals Inc. recently presented updated phase 2 data at the 2023 European Association for the Study of the Liver…
In March 2023, the European Commission (EC) approved Pombiliti (cipaglucosidase alfa) for the treatment of adult patients living with late-onset Pompe disease. Just three months later in June, another…
American consumers have access to the most advanced pharmaceutical systems in the world. Recent medical and technical advances seem to support that reputation. According to Medical Xpress, a Swiss…
According to a story from International Business Times, a French couple encountered an unpleasant skin condition as a result of a pest infestation in their home. Their case was reported…
The FDA has approved Rystiggo, a developed by UCB in Brussels, to treat two types of myasthenia gravis. The approval was based on the results of the Phase III…
14 touchdowns. 11.6 yards per carry (on average). 1st Team All-Conference. These accolades show how hard Luke Winterbottom, an all-league running back for Plymouth Whitemarsh High School, worked during…
The Orphan Drug Act of 1983 was implemented after the U.S. Food and Drug Administration observed drug development issues in the rare disease space. Rare diseases often have smaller…
On June 21, 2023, the Rare Disease Legislative Advocates (RDLA) hosted its monthly webinar. These webinars help provide updates to the rare disease community on legislation and other policy initiatives…
It looked as if the fate of Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy was pretty much sealed until an FDA official intervened. This information was provided by…
When it comes to treating interstitial cystitis, doctors may take a multipronged approach: physical therapy, bladder distention, surgery, nerve stimulation, oral medications. But people with this condition lack therapies…
The U.S. Food and Drug Administration offers various designations to drugs and drug developers to both incentivize medical advancements in the rare disease space and get treatments in the…
In 2015, rare disease stakeholders in North Carolina established the first Rare Disease Advisory Council (RDAC); the National Organization for Rare Disorders (NORD) explains that an RDAC is an…
As recently reported by Forbes, the U.S. Food and Drug Administration (FDA) approved Leqembi (lecanemab) for Alzheimer’s disease earlier this week. Leqembi is a humanized monoclonal antibody that targets…
In late June 2023, global biopharmaceutical company UCB announced that the U.S. Food and Drug Administration (FDA) approved RYSTIGGO (rozanolixizumab-noli) for adults living with AchR or MuSK antibody positive…
Our genes dictate our health in many ways. When working properly, genes encode for the production of various proteins and enzymes throughout the body. So when genes become mutated,…
As of 2020, there were about 241 million cases of malaria occurring on a global scale with a death toll of 627,000. These cases are most common in areas…
According to a recent article in the Pharma Times, E02463 is a therapy for indolent or slow growing, not yet problematic non-Hodgkin B cell lymphoma (iNHL). Enterome, its developer,…
According to a story from statnews.com, a new documentary film from Elle Brooks-Tao titled "I'm Not Crazy, I'm Sick" delves into the stories of three people living with long-term Lyme…
According to a story from BioPharma Dive, Apellis Pharmaceuticals will now join the ranks of pharma companies that have seen an investigational therapy being developed for amyotrophic lateral sclerosis falter…
If you’ve been there, then you know. Patients experiencing cancer, especially the rare aggressive types such as pancreatic cancer, and requiring targeted treatment for the disease may soon be…
Uplifting Athletes, a national nonprofit organization that harnesses the power of sport to support people impacted by rare diseases, celebrated 20 years of Lift for Life with Penn State University…
According to a story from healio.com, an oral medication being evaluated in a phase 2 study was able to increase annualized height velocity in children living with growth hormone deficiency,…
The five-year rate of survival for pancreatic cancer is at 12% having the highest mortality rate of any major cancer. Defence, an aptly-named biopharmaceutical company in Vancouver, B.C. recently…
In January 2023, 23-year-old Angel Anthony Cortez was admitted to the hospital. His health had rapidly deteriorated over the previous days and his family was concerned. Following his admission,…
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