Sedor Pharmaceuticals, a partner of Ligand Pharmaceuticals, has recently received FDA approval for SESQUIENT, its status epilepticus treatment. It has been cleared for the treatment of both adult and pediatric…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
According to a story from pharmalive.com, the drug company Calliditas Therapeutics will plan to seek approval for its therapy Nefecon from the US Food and Drug Administration (FDA). This approval…
According to a story from Cancer Network, the European Commission has recently approved a two-part combination treatment for advanced, homologous recombination deficiency (HRD)-positive ovarian cancer. This first-line maintenance treatment consists…
According to Biospace, Allogene's trial of ALLO-715 for relapsed/refractory multiple myeloma was marred by a patient death. Data from this trial is slated to be presented in December at the…
People with hereditary and acquired angioedema have unpredictable attacks of swelling/inflammation/pain, most of which can be managed with rescue meds like Firazyr, Berinert, Kalbitor, and Rhucin, plus a prescribed med…
The entire world has been waiting for a vaccine for COVID-19 since the beginning of the pandemic in March. In response, a number of pharmaceutical companies have entered the race…
Children's Craniofacial Association (CCA) has posted tips for transitioning to adulthood for those with craniofacial differences. We've summarized some of their recommendations, but you can read the full article here.…
According to a story from Newswise, trial results released in the journal PLOS Medicine suggest that the drug vamorolone could provide an enhancement in the effectiveness of corticosteroid therapy in patients…
According to an article in Biospace, CureVac of Boston, MA, with headquarters in Tübingen, Germany, just announced interim results from its Phase 1 study evaluating CVnCoV, a vaccine candidate against…
CymaBay Therapeutics, a biopharmaceutical company based in Newark, CA, develops and provides access to novel therapies with a recent focus on primary biliary cholangitis (PBC). The company is currently developing…
The FDA has recently granted the Rare Pediatric Disease (RPD) designation to AMO-02, a treatment for congenital myotonic dystrophy. As there is currently an unmet medical need for this condition,…
DNA sequencing has become part of our lives - but what is it? For one thing, it is a big business. In 2003, approximately $3billion US found its way into…
According to a story from GlobeNewswire, the biopharmaceutical company Reata Pharmaceuticals, Inc., recently announced that year two of its phase 3 clinical trial has been completed. This trial is testing…
BioMarin plans to expand its clinical program for vosoritide, its treatment for achondroplasia. This expansion includes two new phase 2 trials, the first of which will evaluate vosoritide in infants…
As reported by the Human Rights Watch; in Mozambique, a grassroots organization to support people with albinism is using murals to spread awareness and a positive image of the disease.…
Did you know that there are multiple forms of multiple sclerosis (MS)? The most common form of MS is relapsing-remitting, meaning patients go through symptomatic periods and periods of remission.…
According to a story from EurekAlert! a recent study presented at the annual meeting of the American College of Rheumatology describes some of the key clinical feature of chronic nonbacterial…
Astellas Pharma, headquartered in Northbrook, Illinois presents its Be R/Ready program to promote patient-physician communication as well as other valuable and informative resources. Their program centers around patients with relapsed…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
A recent article from the CCA has explained some considerations for mask types that you may not have thought about. Some of these are discussed below, but you can read…
According to Newswise; in a new review of studies which looked at 36 studies on the COVID-19 virus found that almost one in five people with COVID-19 only show gastrointestinal symptoms,…
In late October, the American Society of Nephrology (ASN) held the ASN Kidney Week 2020 Reimagined. During the conference, biopharmaceutical company FibroGen shared two posters (abstracts PO2625 and PO2626) regarding…
by Danielle Bradshaw from In The Cloud Copy Bone grafts, although very helpful, can result in hard to treat infections and typically result in the patient needing to be put…
Medical professionals are looking towards patient-reported outcomes more and more as a source for research. In fact, a study titled "Patient reported impact of Charcot-Marie-Tooth disease: protocol for a real…
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