Claire Richmond can be considered a very experienced patient. It took Claire nineteen years to receive a diagnosis for her rare disease. During that time, she had been dismissed…
In the past, some drug developers were not incentivized to develop treatments for those within the rare disease community, considering the smaller patient size. However, the FDA created the Orphan…
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…
Amyotrophic lateral sclerosis (ALS) is a rare, neurological disorder that is characterized by the progressive death of nerve cells and muscle weakness. As time passes, many affected individuals experience difficulty…
St. Jude Children's Research Hospital has recently announced that its team of scientists have established a registry for pediatric melanoma. The goal is to use data from this registry for…
According to a story from GlobeNewswire, the gene therapy company LEXEO Therapeutics has recently announced that its investigational gene therapy LX2006 has been granted both Orphan Drug designation and Rare…
Unfortunately, many molecules which drive cancer development and growth are considered "undruggable." In short, this means that drugs are unable to bind to or address these molecules due to size,…
Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR), according to NCBI, are sequences that replicate and infect bacterial cells. CRISPR has been in the news recently, but this time it has…
In patients with rare or underserved conditions, it can sometimes be difficult to receive effective treatment. For example, chemotherapy, the standard-of-care for Langerhans cell histiocytosis (LCH), is only effective in…
Adhik is alive today thanks to the skill and generosity of the Amirita Hospital in India and others who contributed to the cost of using the highly regarded, but…
BioMan Pharmaceuticals has just announced that vosoritide has just received a positive opinion from the Committee for Medicinal Products in Europe, which means marketing authorization is recommended. Further, it has just…
The FDA grants designations, such as the Rare Pediatric Disease and Orphan Drug designations, to aid in the drug development process. Recently, LEXEO Therapeutics has received these two designations for…
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…
In a story for the Urology Times, a panel of experts on the treatment of non-muscle invasive bladder cancer that is not responsive to Bacillus Calmette-Guérin (BCG) (which is commonly…
In a video posted on Healio, Dr. Lorenzo D'Antiga from the Pappa Giovanni XXIII Hospital talked about the findings from a recent clinical study in which the drug odevixibat (also…
Medical professionals have been searching for a viable treatment for Parkinson's disease for a long time. While there have been advancements and new therapies created, many patients are still looking…
Medpage Today ran the entertaining story of the fabled crow who quenched his thirst by tossing stones in a pitcher of water to raise the water level. Prior to…
On June 22, 2021, PharmaTimes Online reported that the European Commission (EC) granted conditional approval to Koselugo (selumetinib), an orally administered therapy, for use in pediatric patients with neurofibromatosis…
Some good news was reported recently in a Biospace article. Dana-Farber Cancer Institute and Harbour BioMed have agreed to combine resources and co-develop novel biotherapies for oncology and immunological…
As reported in Biospace; a Phase 2 study by Bristol-Myers Squibb and Acceleron Pharma Inc. earned itself recognition as among the top six abstracts received by the Presidential Symposium of…
Orphan Drug designation is a status granted to drugs or biologics intended to treat patients with rare conditions, defined as those affecting under 200,000 Americans. According to Pulmonary Hypertension News,…
Until recently, multiple myeloma patients were reliant upon treatment with immunomodulatory drugs, anti-CD38 antibodies, proteasome inhibitors, and autologous (patient-derived) stem cell transplants. According to a recent article in CancerConnect, patients…
The National Medical Products Administration (NMPA) of China has recently approved surufatinib for drug registration for the treatment of advanced pancreatic neuroendocrine tumors (pNETs). HUTCHMED Limited recently announced the news,…
Patient Worthy signed up to cover several sessions at the Huntington's Disease Society of America's (HDSA) 36th Annual Convention, which was held virtually this year. This event features informative presentations…
The pressure was on. Researchers had been studying Alzheimer’s for decades. Every morsel of discovery was hailed as moving closer to a cure and results were always expected soon…
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