At the end of June 2021, pharmaceutical company Harmony Biosciences Holdings, Inc. ("Harmony") shared the initiation of a Phase 2 clinical trial via news release . During the trial, the…
In the European Union (EU), Orphan Drug designation is granted by the European Medicines Agency (EMA). The status is given to drugs or biologics intended to treat, prevent, or diagnose…
In the United States, the Orphan Drug Act was developed to expedite the development and review of drugs or biologics intended to treat rare or life-threatening conditions. For the purpose…
The FDA has recently granted their approval to a Phase 2 trial of FHL-301, a treatment for those in the earlier stages of Parkinson's disease. Forest Hills Lab will conduct…
The primary cause of death in melanoma patients is widespread metastases. Currently, there is no cure in cases of relapse or in advanced stages. A recent article in Science…
The European Commission recently approved of Enspryng for neuromyelitis optica spectrum disorder (NMOSD). Now, European patients have a new treatment option. This one is especially useful as it can be…
According to a story from Parkinson's News Today, a small-scale study suggests that Virtual Reality (VR) gaming could be a useful and effective form of exercise for people living with…
As reported in World Pharmaa Today, Valneva SE is a specialty vaccine company in the business of the creation and distribution of prophylactic vaccines that treat infectious diseases with serious…
About a month ago, from June 23-26, The Immune Deficiency Foundation hosted the 2021 Primary Immunodeficiency Conference. The event was the largest gathering of primary immunodeficiency (PI) patients across the…
In a video conversation recorded for Cancer Network, Kristie L. Kahl and Javier L. Munoz, MD, MS, FACP discussed treatment for relapsing/remitting follicular lymphoma and the importance of the sequencing…
Have you ever heard of Bachmann-Bupp Syndrome (BABS)? If not, don't worry, you're not alone. In fact, BABS was only first discovered over the past few years. According to Medical…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
The very first HAE symptom I had brought me to the operating table. Unnecessarily. It was summer, with holidays and grandmother's birthday party- a big family reunion in the garden.…
Following several Patient Worthy articles on the subject earlier this year, Fierce Biotech recently published news that Biogen is preparing to release the Phase III VALOR trial drug, tofersen,…
If you could be vaccinated against serious conditions via nose drops, would you take that option? Well, it could one day be a possibility. According to New Atlas, scientists from…
According to a story from USA Today, Mark Hoppus, bass player for the iconic pop-punk band Blink-182, was recently diagnosed with a rare cancer: diffuse large B-cell lymphoma. His cancer…
According to a news release from late June 2021, biopharmaceutical company Pharming Group N.V. ("Pharming") completed enrollment for a Phase 2/3 clinical trial evaluating leniolisib for patients with activated phosphoinositide…
OncLive recently interviewed Daniel Pollyea, MD, MS, about the current treatment landscape for higher-risk myelodysplastic syndromes (HR-MDS), along with exciting new data from a Phase 2 trial of pevonedistat plus…
Researchers from Boston University and Boston Medical Center have recently uncovered that base editing is able to correct the gene mutation which causes alpha-1 antitrypsin deficiency (A1AD). This condition impacts…
While myasthenia gravis (MG), an autoimmune neuromuscular disorder, can be treated, there is a smaller subset of patients who are treatment-refractory. Those whose condition is not easily treated are thought…
The name of the organization is Disease InfoSearch. It is not only a resource for credible information but guides patients and families to valuable support groups and participation in…
In the company's Phase 2 proof-of-concept clinical trial, biopharmaceutical company Harbour BioMed ("Harbour") sought to understand the impact of batoclimab (HBM9161) on patients with generalized myasthenia gravis (gMG). The…
If you have ever donated to the American Red Cross, you've probably been receiving a rash of calls and emails asking for donations. I know I have. This is because…
In the past, Crysvita was approved for subcutaneous administration by a physician for patients with X-linked hypophosphatemia (XLH). This injection could be both invasive and time-consuming. However, according to the…
This question is what the doctors at Massachusetts’ Cambridge Hospital were trying to determine. According to a recent article in the New York Times, a twenty-two-year-old man was rushed to…
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