According to a press release from Inovio Pharmaceuticals, the biotechnology company has officially dosed the first patient in its phase 1/2 clinical trial, which will test the company's investigational therapeutic…
According to a story from Newswise, the ongoing coronavirus/COVID-19 pandemic has become the subject of wild speculation. In our divided nation, many people have chosen to hear what they want…
According to a report in BioPharma Dive, Brainstorm Therapeutics, a New York biotechnology company, said that while its NurOwn therapy for amyotrophic lateral sclerosis (ALS) appeared to have a…
In late November, RNAi therapeutics company Alnylam Pharmaceuticals ("Alnylam") announced that its therapy OXLUMO (Lumasiran) received approval from the European Commission (EC) for both pediatric and adult patients with…
According to a story from Newswise, a collaborative research team from the University of California San Diego School of Medicine and the Rady Children's Institute for Genomic Medicine will be…
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…
Dilated cardiomyopathy is a heart condition caused by a mutation in the RBM20 gene. This heart condition is unique because it can affect young adults. These individuals are at an…
According to a press release from the biopharmaceutical company Sanofi, the company's investigational treatment avalglucosidase alfa will have its Biologics License Application (BLA) evaluated by the US Food and Drug…
PMDD Premenstrual dysphoric disorder (PMDD) is a psychological condition which cyclically occurs with the menstruation cycle. Unfortunately, because the symptoms of this condition can mirror many mental health diagnoses, it…
A recent analysis has shown that eosinophilic gastritis and eosinophilic duodenitis are severely undiagnosed or misdiagnosed as another GI condition. Allakos Inc. has recently reported the results of this study,…
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…
Research on myalgic encephalomyelitis, or chronic fatigue syndrome (ME/CFS), has developed in recent years; however, until now there has still been a gap in the literature regarding loneliness. This condition…
Solid Biosciences has just reported that the FDA has finally lifted the hold they had placed on their Phase I/II trial for Duchenne muscular dystrophy (DMD). This trial is called…
According to a story from Fierce Pharma, the drug company Alnylam Pharmaceuticals has seen its investigational therapy lumasiran (marketed as Oxlumo) recently gain approval for use in the EU as…
According to a story from PR Newswire, the pharmaceutical and diagnostics company The Menarini Group recently announced that its drug tagraxofsup (marketed as Elzonris) has a received a positive opinion…
Although the American College of Asthma, Allergy and Immunology (ACAAI) Annual Scientific Meeting has wrapped up for this year, there is still a ton to learn from the presentations…
In mid-November, The Assistance Fund (TAF) announced that they were beginning a copay assistance program for those with acromegaly. For nearly 11 years, TAF has worked to assist patients…
According to a press release from the biopharmaceutical company Sanofi, the Biologics License Application (BLA) for its drug avalglucosidase alfa has been accepted by the US Food and Drug Administration…
According to a story from Charcot-Marie-Tooth News, patients living with Charcot-Marie-Tooth disease have reported in a recent survey that their symptoms of pain and decline in mobility increased during the…
Early last week, Agios Pharmaceuticals ("Agios") announced that its treatment Mitapivat received Orphan Drug designation. This orally administered therapy is a pyruvate kinase R (PKR) activator. As Agios has…
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…
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.…
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 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…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
