Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.
If I told you that tiny bubbles could be used to more effectively treat cancer, would you believe me? Well, in the case of neuroblastoma, that might be correct. According…
On July 28, 2021, biopharmaceutical company Thetis Pharmaceuticals ("Thetis") shared that its small molecule Resolvin E1 (RvE1) therapy, TP-317, received Orphan Drug designation from the FDA. While TP-317 has…
In the United States alone, an estimated 300,000 people have moderate-to-severe chronic idiopathic urticaria (CIU), or chronic hives which are either recurrent or last for over 6 weeks, which…
Orphan Drug designation is a status granted by the FDA to drugs or biologics intended to treat patients with rare conditions. In this case, the FDA defines "rare" as…
Previously, biopharmaceutical company XyloCor Therapeutics ("XyloCor") performed the first portion (dose-escalation) of the Phase 1/2 EXACT clinical trial evaluating its investigational treatment XC001 (encoberminogene rezmadenovec) for patients with refractory…
According to a relatively recent news release from biopharmaceutical company Dicerna Pharmaceuticals, Inc. ("Dicerna"), interim data is now available from a Phase 1 clinical trial evaluating belcesiran for patients with…
According to a July 27 news release from biopharmaceutical company Day One Biopharmaceuticals ("Day One"), the company's therapeutic option DAY101 received Rare Pediatric Disease designation within the United States. Altogether,…
In a news release from July 21, 2021, biotechnology company Genentech shared that its therapy Venclexta (venetoclax), in conjunction with azacitidine, received Breakthrough Therapy designation from the FDA. Overall, the…
Sometimes, clinical trials do not go the exact way that is expected. According to Targeted Oncology, this is what happened in a Phase 1b clinical trial evaluating RVU120 for…
Unfortunately, there are a great deal of inequities within the healthcare system. Health inequity occurs when groups are treated differently, offered different or worse care, or have a lower…
According to a press release from July 15, 2021, the Chinese NMPA recently approved an Investigational New Drug (IND) application submitted for CAN106, a potential therapeutic option for patients with…
Near the end of June 2021, pharmaceutical company Crinetics Pharmaceuticals, Inc. ("Crinetics") shared via news release that the first patient had been dosed in the Phase 3 PATHFNDR-1 clinical trial.…
Medical studies are crucial players in the journey to develop new and more efficacious treatment options for patients with rare diseases. On June 10, 2021, Vertex Pharmaceuticals Incorporated ("Vertex") shared…
In the past, researchers have shown that acute myeloid leukemia (AML) may be driven by certain genetic mutations. For example, some mutations might cause the abnormal or excess production of…
Genetic editing has the potential to address and treat a variety of genetic conditions. Recently, researchers explored gene editing as a potential therapeutic option for patients with facioscapulohumeral muscular dystrophy…
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…
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…
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 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…
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…
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…
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…
Clinical trials can be crucial in determining potential treatment options for a variety of conditions. According to Pharma Biz, biopharmaceutical company Nordic Nanovector ("Nordic") recently shared promising data from the…
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