Recently, twenty-two year old Jordan Ray of Wellington, Florida spoke with a reporter from The Palm Beach Post, recounting how her life changed in April 2015 during a softball…
Medical professionals have already discovered a number of genes that play a role in pulmonary arterial hypertension (PAH), but according to LabRoots, they have found a new one. The ABCC8…
Researchers have announced their identification of a specific gene mutation that can lead to atypical hemolytic uremic syndrome (aHUS). This gene is called DGKE and it is not a part of…
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…
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…
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…
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…
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…
According to a story from Charcot-Marie-Tooth News, an experimental HDAC6 inhibitor called CKD-504 appeared to restore myelination in mouse and cell models of Charcot-Marie-Tooth disease type 1A in a recent…
Dr. Naveen Pemmaraju recently interviewed with Targeted Oncology and discussed the many advances in myeloproliferative neoplasms (MPNs) treatment as well as the role of social media during the pandemic.…
Chronic pain is a common problem in the United States; it is estimated that 25.3 million people in the US experience chronic, severe pain every day. Within these millions of…
Turning 18 means many things: the right to vote, buy a lottery ticket, join the military, and even go skydiving. That transition into independence gives freedom, but also newfound responsibility.…
Dr. Richard Furie at New York’s Feinstein Institutes recently interviewed with MedPage Today. Dr. Furie discussed the prognosis for lupus nephritis and belimumab (Benlysta), a recently-tested drug that appears…
This year, thanks to COVID-19, many conferences have been held online. These play a crucial role in spreading data, facilitating conversations, and determining potential treatment options for patients with…
I'm sure you have heard about Zika virus before, but what about Chikungunya? Both are rare mosquito-borne diseases that can cause severe neurological problems. But now, researchers also believe…
The care of a loved one with a debilitating condition often falls to their mother, but fathers, siblings or spouses also may take on that role. Even if there is…
Graves' Disease Awareness Month is recognized in July of each year. As part of this year's awareness campaign, the nonprofit organization Prevent Blindness and Horizon Therapeutics collaborated to create the…
According to an article from BioTech 365, data from the Phase 3 INVICTUS trial and Phase 1 dose-escalation study, which evaluated QINLOCK as a treatment for gastrointestinal stromal tumors, was…
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…
Recently, clinical-stage biopharmaceutical company Aura Biosciences announced a Phase 2 clinical trial to determine the safety, efficacy, and tolerability of AU-011 for patients with choroidal melanoma. Currently, Aura Biosciences…
According to Ankylosing Spondylitis News, data from the Phase 3 PREVENT clinical trial highlights Cosentyx (secukinumab) as an effective treatment for patients with non-radiographic axial spondyloarthritis (nr-axSpA), as well as…
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