Recently, the Janssen Pharmaceutical Companies of Johnson & Johnson reported that they submitted a New Drug Application (NDA) for intravenously administered UPTRAVI (selexipag). The therapy is designed to treat…
A recent article in The American Journal of Managed Care (AJMC) heralds the approval of a third CAR T-cell therapy, called Tecartus. Kite Pharma’s cell-based gene therapy is the first…
According to ONCLive, ripretinib, designed to treat patients with advanced gastrointestinal stroll tumors (GIST), was given Priority Review status in China by the National Medical Products Administration. This status…
The month of August is recognized as Stevens-Johnson Syndrome Awareness Month. The goal of this event is to elevate awareness about Stevens-Johnson syndrome among the general public and in the…
A recent article in The American Journal of Managed Care (AJMC) heralds the approval of a third CAR T-cell therapy, called Tecartus. Kite Pharma’s cell-based gene therapy is the first…
Last month, at the American Academy of Dermatology (AAD) Virtual Annual Meeting, Dr. Dedee F. Murrell, MD, explained the efficacy of rilzabrutinib for treating pemphigus. According to MDedge, many…
In a recent webinar hosted by the National Organization for Rare Disorders (NORD), a number of speakers discussed an up and coming project spearheaded by the organization: the Rare Disease…
In a recent press release, biopharmaceutical company Fortress Biotech announced that their study regarding ATP7A targeted next generation DNA sequencing for Menkes disease was published in Molecular Genetics and Metabolism…
In a recent webinar hosted by the International Waldenström's Macroglobulinemia Foundation (IWMF) and held on July 22nd, 2020, Dr. Jeffery Matous from the Colorado Blood Cancer Institute gave an in-depth…
According to a story from Medscape, the US Food and Drug Administration (FDA) has recently approved a new oral solution as a treatment for symptoms associated with narcolepsy, a rare…
According to a story from Globe Newswire, the biopharmaceutical company Ovid Therapeutics and the University of Connecticut School of Medicine (UConn) have announced a new license agreement and research collaboration…
In order to better understand rare conditions and diseases, researchers sometimes create registries and biobanks full of resources. Recently, the Solve ME/CFS Initiative (Solve M.E.) announced the launch of…
If you're part of the rare disease community, then you probably are not a stranger to explaining your condition to others. But think about it. Has this ever been…
In a recent press release, Knopp Biosciences announced that their drug therapy KB-3061 was granted Rare Pediatric Disease Designation to treat patients with KCNQ2 epileptic encephalopathy (KCNQ2-EE), a rare…
For a lot of us, self-care is difficult. This is for a lot of reasons, some of which might be guilt, not enough time, putting others before ourselves, not sure…
According to a story from Buzzfeed News, a recent test of an experimental COVID-19 vaccine being developed by Moderna Therapeutics demonstrated that it was able to produce antibodies in all…
According to a story from meaction.net, Dr. Anthony Fauci was recently asked a question about what was being done to address so-called COVID-19 "long haulers," and their potential risk of…
Each year, June 13th is celebrated as Gastrointestinal Stromal Tumors (GIST) Awareness Day. The ultimate goal of awareness days for rare diseases is to help inform both the general public…
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…
At the end of June, the National Hemophilia Foundation (NHF) released a warning to patients with hemophilia regarding AT132, an investigational gene therapy candidate. Developed by Audentes Therapeutics, AT132 uses…
According to a recent press release, biotechnology company Cancer Prevention Pharmaceuticals (CPP) submit a New Drug Application (NDA) for accelerated approval of their drug therapy CPP-1X/sul to treat familial adenomatous…
Without treatment, spinal muscular atrophy (SMA) is the top cause of infant or child mortality before age 2. As a result, early detection is crucial in improving patient outcomes. But…
This morning, SynAct Pharma AB announced in a press release that they began a Phase II clinical trial to determine the efficacy and safety of AP1189 on treating patients with…
According to a story from PR Newswire, the US Food and Drug Administration (FDA) has recently approved a new treatment for Dravet syndrome, a rare disorder that causes seizures and…
It amazes me how quickly the numbers change each time I write about COVID-19, the novel coronavirus declared a global pandemic in March 2020. As of the afternoon of…
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