Updates from the Phase 3 clinical trial called FIGHT-302 were just released at the 2021 European Society for Medical Oncology World Congress on Gastrointestinal Cancer. The trial is comparing two…
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…
According to a story from Market Watch, the biopharmaceutical company Ultragenyx Pharmaceutical, Inc. recently announced that it was given Orphan Drug designation from both the US Food and Drug Administration…
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…
According to a recent article in Genetic Engineering News, findings by scientists at the University of Boston’s School of Medicine and Massachusetts General Hospital (MGH) have uncovered two plausible…
According to a video from OncLive, Dr. John P. Leonard from Weill Cornell Medicine discussed the role of PI3K inhibitors in the treatment of follicular lymphoma, a rare cancer. While…
According to a press release published on Globe Newswire, the biopharmaceutical company Saniona has announced recently that its investigational therapy Tesomet has recently earned Orphan Drug designation from the US…
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…
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…
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,…
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…
Acknowledgment: This story is sponsored by Takeda and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing relevant, vetted and valuable information to the…
From July 2-3 and 8-10, 2021, the European Crohn's and Colitis Organisation (ECCO) held its 2021 virtual Annual Congress (ECCO'21). During the Congress, many discussed new research and trends…
Access to treatment is an issue for patients across the world, especially for those with rare diseases. Laurie Young, who was diagnosed with type 2 spinal muscular atrophy (SMA) at…
According to a story from MarketWatch, the biotechnology company Cyclo Therapeutics announced that is has proceeded with the enrollment for its phase 3 clinical trial. This trial will evaluate an…
In a review posted on dovepress.com, monoclonal antibodies have established themselves as an important tool in the management of multiple sclerosis in both the relapsing-remitting form and the progressive form.…
The International Waldenstrom's Macroglobulinemia Foundation (IWMF) recently held the latest webinar in its Global Education series on July 9, 2021. This webinar was a summary of research presented at the…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is…
KRYPOLIS (carfilzomib) recently received conditional approval in China for the treatment of relapsed/refractory (R/R) multiple myeloma. Licensed by BeiGene, this drug is indicated for adults with R/R multiple myeloma who…
Spinal muscular atrophy is rare progressive muscular disease that over time, erodes a patient’s movement. Without early intervention, the condition erodes the young patients ability to breathe and even swallow.…
Accessing treatments can be difficult, especially if one is impacted by a rare disease. In fact, only 5% of rare diseases have an FDA-approved treatment. Because of this, many patients…
According to a July 13 news release, biopharmaceutical company Kedrion Biopharma ("Kedrion") recently treated its first pediatric patient within the Phase 3 KIDCARES10 clinical trial. Within the trial, Kedrion…
Beijing, China: According to an article in a sister publication of Sixth Tone, a woman living in China who has been treated during the past fourteen years for paroxysmal nocturnal…
Thanks to advances in immunotherapy, liver cancer (Hepatocellular carcinoma-HCC) has seen a rise in treatment options for patients in advanced stages of the disease. HCC is one of the…
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