by Danielle Bradshaw from In The Cloud Copy A recently performed study has demonstrated that it is possible to use tablets to screen patients that have chronic lung conditions -…
Edgewise Therapeutics has announced their plan to initiate a Phase 1 trial of EDG-5506, their small molecule therapy for Becker muscular dystrophy. It will take place at Worldwide Clinical Trials…
by Lauren Taylor from In The Cloud Copy Nonalcoholic fatty liver disease, or NAFLD, and nonalcoholic steatohepatitis, or NASH, are two conditions affecting the liver that plague millions of Americans…
Rhythm Pharmaceuticals, based in Boston, MA, is a biopharmaceutical company focusing on the development of therapies to treat rare genetic diseases that cause obesity. Until now, there have not been…
IntraBio has recently launched an extension study of their Phase II trial of IB1001, a treatment for Niemann-Pick disease Type C. This decision was made shortly after analyzing the positive…
As reported in the BBC; Aaron Bradley is concerned that returning to work will endanger his son’s health. This follows the Scottish governments decision to move his toddler off of…
Sierra Oncology has recently released two abstracts that will be presented at the 62nd American Society of Hematology (ASH) Annual Meeting. The meeting is planned for December 5-8. Both abstracts…
According to a story from Newswise, the genome editing company Intellia Therapeutics has recently announced that the first patient has been dosed in its phase 1 trial, which will test…
From November 5 through 9th, the American College of Rheumatology (ACR) held their ACR Convergence 2020. Though the annual meeting took place online, it was still rife with insights into…
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.…
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