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…
The month of July is recognized as Sarcoma Awareness Month, a time of the year for spreading awareness about sarcoma among the general public and in the medical community. An…
Often misdiagnosed as other conditions such as Rett syndrome or cerebral palsy, DDX3X mutations cause both cognitive and physical difficulties. Since only an estimated 700 patients have been diagnosed…
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…
July is recognized as Juvenile Idiopathic Arthritis Awareness Month. Though arthritis is often considered an illness that is associated with old age, it is estimated that around 300,000 children in…
Two new reports of Guillain-Barré syndrome (GBS) have been documented in response to the AstraZeneca COVID-19 vaccine. Previously, published papers have documented 4 cases in England and 7 cases in…
Newborn screening is important to early diagnosis and intervention. With some conditions, every second counts. Catching a rare disease as soon as a child is born could lead to much…
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…
Aurinia Pharmaceuticals has just announced that Otsuka Pharmaceutical Europe Ltd. (OPEL), their licensing partner, has just filed a Marketing Authorization Application for an investigative therapy for lupus nephritis (LN) called…
I-Mab has just announced China's Center for Drug Evaluation has officially approved the new drug application for felzartamab as therapy for systemic lupus erythematosus (SLE). That means that a Phase…
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 very first corneal allogenic intrastromal ring segment surgery (CAIRS) for keratoconus has just been completed in Australia. It was completed at Queensland Eye Institute by David Gunn. This procedural…
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…
Anybody who knows me knows that Jeopardy! is one of my favorite shows (Rest In Peace, Alex Trebek). Of course, that doesn't mean that the show never messes up or makes…
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…
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…
Researchers have just announced that they have completed enrollment for a new Phase 3 trial for patients with transthyretin-mediated (ATTR) amyloidosis who have heart disease. This trial is examining a…
According to a story from nbcnewyork.com, A resident of Sussex County, New Jersey has recently tested positive for a rare infectious disease called Jamestown Canyon virus, or Jamestown Canyon encephalitis.…
Strongbridge Biopharma has just announced that the FDA has decided to review the New Drug Application for RECORLEV, a therapy for endogenous Cushing's syndrome. The target action date is January…
The Rare Disease Legislative Advocates (RDLA) webinar for the month of June took place on June 17, 2021. This month's webinar provided updates on a variety of legislative topics that…
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,…
As reported by Oncology Nursing News, gunagratinib (ICP-192) was recently granted Orphan Drug designation by the FDA. Altogether, this treatment is being evaluated as a potential treatment for patients…
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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