On January 27, 2022, The EveryLife Foundation for Rare Diseases, in partnership with the Biotechnology Innovation Organization (BIO), National Health Council, and Pharmaceutical Research and Manufacturers of America (PhRMA), hosted…
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…
At the end of last year, the FDA warned people with hyperkalemia to monitor their diets when it comes to potassium intake. According to an article from the Taipei Times,…
Recently, Chain Drug Review reported that the FDA had approved a generic form of colchicine pills (normally prescribed as Mitigare, Colcrys, or Gloperba) for the treatment of familial Mediterranean fever (FMF).…
According to a recent article, the drug dovitinib has been submitted to the FDA for pre-marketing approval as a third-line therapy for patients diagnosed with renal cell carcinoma (RCC). Renal…
According to a recent article, a new stem-cell-based gene therapy is showing promising results for treating beta (β)-thalassemia in a phase 1 clinical trial. Beta Thalassemia Beta thalassemia is a…
Nonalcoholic steatohepatitis is a liver disease, causing fat to build up in the liver. The resulting inflammation may lead to liver cancer or cirrhosis (scarring) and possible liver failure. As…
Many people living in rural areas of the U.S. must drive a long distance to receive their medical treatments. According to Yahoo News, twenty-nine-year-old Leah Stavenhagen, now living and…
The recent FDA approval of Vyvgart (efgartigimod) for generalized myasthenia gravis (gMG) marks the first drug approval for Argenx. Vyvgart is designed specifically for adults who test positive for…
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…
WKBW Buffalo recently interviewed ALS patients who are advocating for a bill that would grant access to clinical trial drugs during Phase III and before they go through the…
In the European Union (EU), Orphan Drug designation is given to drugs or biologics intending to improve the treatment, diagnosis, or prevention of rare or serious illnesses. To be…
ANCA-associated vasculitis (AAV) generally targets two enzymes, myeloperoxidase (MPO) or proteinase 3 (PR3) located in the granules of monocytes and neutrophils. These enzymes play a critical role in 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 a…
According to a recent article, a new study shows a daily oral dose of Belzutifan may keep patients diagnosed with von Hippel-Lindau disease from needing surgery by shrinking tumors. Von…
After several clinical setbacks, Vertex Pharmaceuticals found promising results in its study of a drug to treat a genetic kidney disease called focal segmental glomerulosclerosis (FSGS). As reported recently by…
Dr. Joshua Sabari, a professor at New York University's Department of Medicine, has recently spoken about the different treatment options for ALK-positive non-small cell lung cancer (NSCLC). Specifically, he discussed…
The FDA grants the Orphan Drug designation to treatments indicated for rare diseases, which are defined as affecting less than 200,000 people in the U.S. This designation is meant to…
According to Dr. Deepak Chellapandian, a Johns Hopkins specialist in bone marrow transplant for rare diseases, the prognosis for his patient was very poor. Jocelyn was born with chronic…
According to DocWireNews, on December 16, 2021, the U.S. Food and Drug Administration (FDA) granted Accelerated Approval to Tarpeyo (budesonide) delayed-release capsules. The therapy is designed to treat adult…
Washington University Researchers readily acknowledge the attributes of sonodynamic therapy (SDT) as a promising therapeutic method to treat hepatocellular carcinoma (HCC). SDT is noninvasive with high tissue penetration, but…
Dr. Richard Stone recently interviewed with Targeted Oncology to discuss challenging acute myeloid leukemia subtypes and a list of available therapies. Dr. Stone was asked to look back at…
Pancreatic cancer so far has been able to escape early detection. When it is discovered, it is usually in an advanced stage and difficult to treat. According to a…
On December 17, 2021, global immunology company argenx SE ("argenx") shared on its website that its therapy VYVGART (efgartigimod alfa-fcab) was approved by the FDA for the treatment of…
Discovering a treatment for a rare disease is challenging and rewarding, especially when it is an extremely rare disease such as perivascular epithelioid cell tumor (PEComa). Biospace recently carried…
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