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…
Pancreatic cancer is one of the deadliest forms of cancer. According to the American Cancer Society (ACS), only about 11% of patients are expected to survive more than five years…
It is very rare that one might say that a car accident saved their life – but Yolanda Carusciello, who lives in Brooklyn, can say just that. Of course, when…
On May 26, 2022, biopharmaceutical company Nurix Therapeutics, Inc. ("Nurix") shared via news release that positive data was available from an ongoing Phase 1a dose escalation study. Within the study,…
The U.S. Food and Drug Administration (FDA) has recently granted approval to tisagenlecleucel, which is marketed under the name Kymriah, for adult patients with relapsed or refractory (R/R) follicular lymphoma…
Fast Track designation is designed to facilitate the development of, and expedite the review of, drugs intended to treat serious conditions and fill unmet patient needs. This designation comes alongside…
Perry Cox is feisty, full of energy, and loves taking part in new activities with her friends. According to her father Griffin, Perry, now five years old, loves dancing and…
From 2010-2013, Abi Phillips embraced the role of Liberty Savage on Hollyoaks, a British fictionalized TV series about the lives of teenagers and their families. The program has since been…
Maria’s message is: “when in doubt, get a biopsy." Ten years ago, Maria Sylvia noticed a vertical streak on her thumbnail that at the time she thought was “cool." She…
Earlier this year, 4D Pharma Plc. ("4D Pharma") experienced a "win" when the FDA approved the company's plans to initiate a Phase 1 clinical trial evaluating MRx0005 and MRx0029 for Parkinson's…
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