The Cambridge Rare Disease Network presents RARE Fest 2020 Engage.Educate.Empower. November 28, 2020 In light of the COVID-19 pandemic, RAREfest20 will be celebrated as a live virtual festival that will…
On November 19, 2020, Research!America hosted an informational webinar titled "From Nobel Winning Science to Next Generation Treatment: Tracing the Path of a Rare Disease Breakthrough using RNAi." This webinar focused…
Thanksgiving is a time of gratitude, family and maybe a little gluttony. It was always my favorite time of the year in high school and college. Now, while I still…
According to a story from PR Newswire, The Autoimmune Registry, Inc. (ARI) has recently released its first ever autoimmune diseases list. This list includes a total of over 150 distinct…
Navigating a Rare Disease in a Pandemic The COVID-19 pandemic has affected everyone, but those already living with a rare disease have unique challenges. With a compromised immune system, they…
We often hear people talking about the stress they are feeling during the holidays, while those in the rare disease community silently think, "You have no idea!" During this season,…
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…
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…
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,…
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.…
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…
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…
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…
United Therapeutics Corporation has recently presented data from the Phase 3 INCREASE trial, which evaluated Tyvaso as a treatment for pulmonary hypertension associated with interstitial lung disease. Results were presented…
According to a story from GlobeNewswire, the company BioCryst Pharmaceuticals, Inc., recently announced that its drug berotralstat (marketed as Orladeyo) will be available in the UK under the Early Access…
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