Last week, biotechnology company Anokion SA ("Anokion") announced the FDA's acceptance of an Investigational New Drug (IND) application. Anokion submitted the IND for ANK-700, a therapy for patients with…
Disclaimer: This blog post has been republished with permission from the author. To visit the original blog site, click here. Written by Tamara Fowler A few weeks ago, during one…
BioMarin has been working to develop treatments for phenylketonuria for 15 years, and it is a cause they are very committed to. That is why they are so excited to…
by Danielle Bradshaw from In The Cloud Copy The University of Manchester’s team of researchers has come up with a new and simple means of monitoring their own electrocardiograms (ECG)…
Lupus Canada and the Lupus Foundation of America have joined together to award the 2020 Lupus Canada Catalyst Award. This grant is intended to fund one year of research that…
Arrowhead Pharmaceuticals recently announced positive twenty-four-week biopsy results from four patients who participated in the first cohort of the Phase II clinical trial of ARO-AAT. ARO-AAT is Arrowhead’s investigational…
by Lauren Taylor from In The Cloud Copy Adrenal insufficiency, also called Addison’s disease, is a condition in which the body does not produce adequate amounts of certain necessary hormones.…
When children have problems at school, it is often linked to something bigger going on in another aspect of their life, whether that is at home or with friends. A…
Recently, researchers sought to better understand the natural history and natural progression of type III spinal muscular atrophy (SMA). According to the AJMC, this study offers more insight into…
Several decades ago, gene therapy seemed to be on target to treat a disease by simply replacing a defective gene with a healthy one. A recent article republished in APNews…
On September 25, biotechnology company Vertex Pharmaceuticals Incorporated ("Vertex") announced FDA-approval of KALYDECO (ivacaftor) for pediatric patients with cystic fibrosis (CF). Specifically, KALYDECO can treat infants between 4-6 months…
Disclaimer: This blog post has been republished with permission from the author. To visit the original blog site, click here. Written by Tamara Fowler When faced with your own mortality,…
by Danielle Bradshaw from In The Cloud Copy Fulcrum Therapeutics, a company that focuses on clinical-stage biopharmaceuticals to improve the quality of life for patients that have rare genetic diseases,…
Recently, research and development (R&D) company Neurophth Therapeutics announced that the FDA granted Orphan Drug designation to its therapy NR082. Also known as rAAV2-ND4 or NFS-01 project, NR082 is…
According to a recent press release, Japan just approved ULTOMIRIS (ravulizumab) for patients with atypical hemolytic uremic syndrome (aHUS). Developed by biopharmaceutical company Alexion Pharmaceuticals ("Alexion"), ULTOMIRIS represents a…
Recently, the European Medicines Agency (EMA) recommended Olumiant (baricitinib) for the treatment of adult patients with moderate to severe atopic dermatitis. Currently, Olumiant is approved for the treatment of…
by Lauren Taylor from In The Cloud Copy Spinal muscular atrophy or SMA is a genetic disease that affects the central and peripheral nervous systems, as well as voluntary muscle…
According to a story from GlobeNewswire, Exagen, Inc. recently launched a testing panel called AVISE® Vasculitis AAV, which is designed to help physicians get fast and accurate data for patients…
For many years, researchers linked obesity to a number of other health conditions. Now, Taiwanese researchers identify a new cause for concern: worsening health outcomes for patients with ankylosing…
In a recent press release, clinical-stage pharmaceutical company Crinetics Pharmaceuticals announced that their investigational drug candidate, CRN04777, received Rare Pediatric Disease designation from the FDA. The therapy is designed…
Disclaimer: This blog post has been republished with permission from the author. To visit the original blog site, click here. Written by Tamara Fowler Where do I begin? Thank you…
Happy Friday! September is a packed with rare disease awareness, as we've been featuring all month long. We wanted to make sure to draw attention to Dystonia Awareness Month, which…
In a recent press release, precision therapy company Blueprint Medicines Corporation announced positive results from two clinical trials: EXPLORER and PATHFINDER. The trials examined the safety, efficacy, and tolerability…
Knowing how to help treat your disease and being able to afford those needs are two different questions. As reported in Newswise, in a study from Harvey L. Neiman Health Policy Institute, researchers…
As sourced from BioSpace: After 44 years of the legendary Jerry Lewis hosting the Jerry Lewis MDA Telethon to bring awareness to this disorder, his tradition will live on in…
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