Misophonia is a rare condition that causes patients to have a severe and involuntary reaction to specific sounds. The cause of the condition is still unknown. A challenge misophonia patients face…
Disclaimer: This blog post has been republished with permission from the author. To visit the original blog site, click here. Written by Tamara Fowler Today was the end of the…
Immune-One Therapeutics has just announced that they have dosed their first acute myeloid leukemia (AML) patient in their Phase 1 trial investigating IO-202. This therapy is an immune inhibitory receptor that works…
by Danielle Bradshaw from In The Cloud Copy Research into the common but rare TP53 mutation - which can be found in many Ashkenazi Jewish people - revealed that it…
Just last week, biopharmaceutical company Mustang Bio announced Orphan Drug designation for its therapeutic candidate, MB-207. This unique lentiviral gene therapy is designed for previously treated patients with X-linked…
According to an article from Charcot-Marie-Tooth News, a study was conducted that discovered three new mutations on the GJB1 gene that result in X-linked Charcot-Marie-Tooth disease (CMTX1). The study, which…
Recently, Blueprint Medicines Corporation ("Blueprint") shared that the European Commission (EC) approved marketing authorization of AYVAKYT (avapritinib). The therapy is designed to treat adult patients with PDGFRA D842V mutated gastrointestinal stromal…
Early last week, biopharmaceutical company MediciNova Inc. ("MediciNova") announced promising results for their therapeutic candidate MN-166 (ibudilast). The company, which published in the findings in Cancer Chemotherapy and Pharmacology, explored…
Written by Paul Pavao The power of a cancer patient's good attitude cannot be over-emphasized. Within the first week of chemo a nurse was willing to tell me, "We can…
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…
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