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…
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…
Jazz Pharmaceuticals has launched an educational program called Find the Right Fit, which aims to spread awareness and help patients with myelodysplastic syndromes (MDS) and secondary acute myeloid leukemia (sAML).…
Happy Friday! Sept. 24 is FH Awareness Day. Familial hypercholesterolemia is a gentic disorder characterized by the inability of the body to remove LDL -- sometimes called "bad cholesterol" --…
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…
Recently, pharmaceutical company Alimera Sciences announced that the first patient was enrolled in the NEW DAY clinical trial to test ILUVIEN as a potential treatment for patients with diabetic macular…
Last month, the annual ESC Congress, headed by the European Society of Cardiology, discussed innumerable treatments and advances in the field of cardiology. During the conference, biopharmaceutical company Kiniksa Pharmaceuticals…
Tyrosinemia is a metabolic disorder which prevents the body from processing the amino acid tyrosine. Without treatment, the disorder can be lethal. There are three different types of tyrosinemia with…
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