Misconceptions exist about many rare diseases. In fact, the efforts of many advocates are focused on education and dispelling these myths. Recently, an article in the Hindustan Times did just…
In a news release from late January 2022, biopharmaceutical company CASI Pharmaceuticals, Inc. ("CASI") shared that the Company's partner, BioInvent International AB ("BioInvent") recently earned an Orphan Drug designation from…
In a February 8, 2022 news release, late-stage clinical company Synthetic Biologics, Inc. shared that the FDA granted Orphan Drug designation to the therapeutic candidate VCN-01. VCN-01 is a therapeutic…
The Memorial Sloan Kettering Cancer Center has recently released skincare guidelines and tips for those living with cutaneous T-cell lymphoma (CTCL). It's important that impacted individuals take good care of…
Researchers are aware that Black cancer patients experience worse outcomes than white cancer patients due to the differences in access to health care. According to a report in Cancer Network,…
It is common practice to treat multiple myeloma patients with a stem cell transplant. In the GENESIS trial, the stem cells were autologous (from the patient’s own peripheral blood or bone…
Currently, there are a number of treatment options for patients with follicular lymphoma. But for patients whose condition has relapsed despite prior treatments, there are very limited therapeutic options. After…
Rare disease patients know what it's like to go without a diagnosis despite enduring symptoms. Doctors often don't recognize the disorders for a number of reasons, whether that's the similarity…
Just about two years ago, Jane Warrimer began experiencing abnormal menstruation. Her period was no longer regular; her bleeding lasted for weeks at a time. After pursuing an IUD, Jane…
According to a press release from the pharmaceutical company Bristol Myers Squibb, the company's CAR T-Cell therapy idecabtagene vicleucel (marketed as Abecma) has been approved by the Japanese Ministry of…
Each year, approximately 1 in every 1 million infants is diagnosed with pediatric myelodysplastic syndromes (MDS), a group of rare disorders characterized by the abnormal or deficient formation of blood…
U.S. News carried an article reporting that in a recent study, researchers have identified minute amounts of cancer (organoids) in fallopian tube tissue. The significance of the discovery is that…
Because there are many obstacles that stand in the way of developing drugs for rare diseases, the FDA grants a number of designations to incentivize companies to create these treatments.…
Orphan Drug designation is granted to drugs or biologics intending to treat, prevent, or diagnose rare diseases or conditions. For the purpose of the designation, “rare” is considered any condition…
In a news release from January 24, 2022, immuno-pharma company AIM ImmunoTech Inc. ("AIM") shared that positive clinical data was now published. The data, which you can find in Clinical Cancer…
Oscar Warrent, a toddler from Norwich, England, has been through a lot in his two years of life. About three months ago, he was diagnosed with rhabdomyosarcoma, a rare form…
Glioblastoma is a highly malignant and aggressive brain cancer. With a 5-year survival rate of under 10%, there is an urgent need for newer and more effective diagnostic and treatment…
According to a recent article, the 2021 American Society of Hematology (ASH) Annual Meeting and Exposition displayed the advances that have been made in the management of follicular lymphoma. Follicular…
A glioblastoma diagnosis can be devastating. Currently, the 5-year survival rate is under 10%. This highly malignant cancer, which forms in the brain and spinal cord, is also somewhat difficult…
Grant and award programs can be crucial in spurring therapeutic discovery and development. According to News Medical, this is what the Follicular Lymphoma Foundation (FLF) aims to do with its…
According to the Cancer Network, a CAR T-cell therapy called C-CAR039 recently earned both Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations from the U.S. Food and Drug Administration.…
What if researchers could change DNA processes within brain tumors to improve overall survival rates and patient outcomes? According to Medical XPress, researchers found that L-methylfolate, in conjunction with the…
Is there a relationship between increased sun exposure and breast cancer risk? At first glance, you might think, “Sure. Sun exposure probably increases the risk.” However, according to an article…
According to Healio, the U.S. Food and Drug Administration (FDA) recently granted Fast Track designation to LioCyx-M004. This therapy, developed by T-cell immunotherapy company Lion TCR, is designed to…
The FDA grants Orphan Drug designation to drugs or biologics intended to treat, diagnose, or prevent rare diseases. For the purpose of this designation, "rare" conditions are those affecting…
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