In January, Harrison Moseby began having shoulder pain that was interfering with his day-to-day life. As a father and a firefighter, Harrison knew that he needed to be functioning as…
Seven-year-old Rydder Ames of Hamilton, Ohio began having troubling symptoms five months ago. His mother, Krista Steven took him to the ER after a week-long headache followed by a…
Seven years ago, Dennis Smith started having extreme back pain. Dennis, 48 at the time, was extremely active. He loved playing football, going to the gym, and riding his bike.…
According to a story from Yahoo News UK, Dr. Paula Hunt had a CT scan earlier this year with alarming results. The scan appeared to show a large tumor in…
Currently, there are an estimated 30 million people within the European Union (EU) who have a rare disease. The “Orphan Medicines” (or Orphan Drug) program was designed to facilitate…
According to a story from Cancer Network, the treatment landscape for myelofibrosis, a cancer of the bone marrow, still has substantial unmet need. An important component of treatment are JAK…
Could a vaccine help treat cancer such as Merkel cell carcinoma (MCC)? Immunomic Therapeutics believes so. Its therapeutic plasmid DNA (pDNA) vaccine, ITI-3000, is designed for those with MCC,…
Currently, clinical-stage oncology company Sumitomo Pharma Oncology, Inc. is working to develop DSP-5336 for patients with acute myeloid leukemia (AML). The company even began recruiting for a Phase 1/2 study…
For most of his life, Jack O’Donnell was as healthy as can be. Even as he headed towards Oklahoma State University, ready to study engineering graphics and design drafting, Jack…
In recent years, there have been advances in the treatment of pancreatic cancer. This is crucial, as the prognosis for this cancer is often poor, and the cancer itself is…
Following the approval of carfilzomib (Kyprolis) for relapsed or refractory (R/R) multiple myeloma (MM) in Japan around two years ago, researchers sought to understand the safety and efficacy of the…
In 2020, Anthony Di Laura and Jackie Cucullo were over-the-moon. The married couple had just learned that Jackie was pregnant with their first child. Anthony and Jackie were thrilled…
In September 2020, Amelia Bradford turned eight months old. Her family loved their fun, wiggly, and happy baby. But then Amelia’s mother Danielle noticed something concerning: a facial bruise around…
Those living with rare diseases or conditions often face difficulty in finding treatment options - or even spurring researchers to make headway within their disease states. To combat this and…
In the United States, the Orphan Drug Act was established to incentivize drug developers and other stakeholders to begin developing therapies for rare diseases. Orphan Drug designation is now granted…
According to Cure Today, the U.S. FDA granted Orphan Drug designation to MB-106, a novel CAR-T cell therapy designed to treat Waldenström macroglobulinemia (WM). In the United States, Orphan…
The American Society of Clinical Oncology (ASCO) Annual Meeting (“ASCO 22”) took place this year from June 2nd through the 6th, 2022. During this Annual Meeting, stakeholders within the…
Have you ever heard of Orphan Drug designation? In the United States, this designation is granted to drugs or biologics that intend to treat, prevent, or diagnose rare diseases…
Sometimes, doctors choose to treat a certain condition with a specific medicine or class of medicines. But what happens when that medicine causes unintended and potentially harmful reactions? For…
When it comes to cancer treatment, it's important to understand what leads to improvements in survival; knowing these things helps to provide the best outcome for the patient. That's why…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare or life-threatening diseases or conditions. Rare conditions are those affecting…
In the United States, the FDA grants Orphan Drug designation to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions. Do you know what makes a…
Currently, no approved treatment options exist for children with pediatric low-grade glioma, the most common brain tumor found in children. However, shares Day One Biopharmaceuticals ("Day One") in a news…
In the United States, a rare disease or condition is one which affects fewer than 200,000 individuals. Alternately, a rare condition in the European Union (EU) is considered one which…
We have been told occasionally that certain phrases we use when speaking with a terminally ill patient may sound insensitive. We have also been told what we should not say…
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