Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.
Recently, IM Therapeutics, a developer of therapies for patients with autoimmune conditions, announced the start of a Phase 1b clinical trial designed to evaluate IMT-002 for type 1 diabetes. Interestingly,…
According to new research, children with juvenile idiopathic arthritis (JIA) are more likely to experience uveitis flares as their arthritis progresses. Medscape notes that researchers wanted to understand how and…
In late October 2020, Homology Medicines ("Homology") announced its development of a new gene therapy program to treat patients with MPS II, or Hunter syndrome. Homology's mission is to meet…
Last week, from October 22nd through the 25th, marked the American Society of Nephrology (ASN) Kidney Week. According to a press release, Synthetic Biotic medicine developer Synlogic, Inc. ("Synlogic") presented…
2326 words (source) vs. 503 words (mine) - 6% match Near the start of October, global biopharmaceutical company Jazz Pharmaceuticals ("Jazz") announced positive safety and efficacy data from their Phase…
Recently, specialty pharmaceutical company Xeris Pharmaceuticals ("Xeris") announced that its injectable treatment XP-0863 received Fast Track designation for the treatment of patients with acute repetitive seizures. Xeris created this treatment…
Early on October 28, 2020, Insmed Inc. ("Insmed") announced that it received Marketing Authorization from the European Commission for ARIKAYCE. This therapy is designed for adult patients with nontuberculous mycobacterial…
In a recent study published in Clinical and Experimental Immunology, researchers determined that patients with high ANCA antibody levels are at an increased risk of having ANCA-associated vasculitis (AAV) relapses. According…
As the medical field develops, more companies are looking to create innovative ways to address cancer treatments. In the case of global pharmaceutical company Daiichi Sankyo Company, Ltd. ("Daiichi Sankyo"),…
In a recent press release, genome editing company Intellia Therapeutics, Inc. ("Intellia") announced its recent MHRA authorization to begin a Phase 1 clinical trial to evaluate its therapeutic candidate NTLA-2001. The…
According to Myasthenia Gravis News, researchers recently determined a new therapeutic target for myasthenia gravis (MG): activating a bile acid receptor called TGR5. By activating this bile acid receptor, researchers…
Many patients with rare conditions know that sometimes it can be extremely difficult to find targeted treatment options. In fact, a large number of rare conditions still have no approved…
On October 22, 2020, BioCryst Pharmaceuticals, Inc. ("BioCryst") announced the publication of data from a Phase 3 clinical trial evaluating berotralstat for patients with hereditary angioedema (HAE). As a…
In early October, the FDA approved an Investigational New Drug Application (IND) for APR-548, a potential treatment for patients with TP53 mutant myelodysplastic syndromes (MDS). According to a press release from…
In mid-October, biopharmaceutical company Zogenix announced that its Dravet syndrome treatment FINTEPLA (fenfluramine) received a positive CHMP opinion. The CHMP is part of the European Medicines Agency (EMA). While…
Earlier this week, the FDA granted Priority Review status to use a combination of Opdivo-Cabometyx as a treatment for patients with renal cell carcinoma (RCC). According to Healio, Opdivo (nivolumab)…
Many people within the rare disease community share a common experience: feeling isolated or misunderstood because of your condition. In some cases, such as conditions causing chronic pain or fatigue,…
At the very core of genetic diseases are - well, our genes. However, each year, an estimated 400,000 infants are born with de novo mutations, or new and spontaneous gene mutations not…
When their son Quinton was diagnosed with late-onset Krabbe disease through newborn screening, Laura and Ryan Nitahara were initially full of questions. What was Krabbe disease? How would this affect their family?…
According to Biotech365, the National Hemophilia Foundation (NHF) recently revised its treatment recommendations for patients with congenital fibrinogen deficiency, or Factor I deficiency. Now, intravenously administered fibryga, developed by…
Recently, clinical-stage biopharmaceutical company Imara Inc. ("Imara") announced that the first patient was dosed in the Phase 2b Forte clinical trial. The trial is evaluating IMR-687, a PDE9-inhibitor, for…
Later this week, from October 21-23, presenters and attendees will be present at the 34th Annual North American Cystic Fibrosis Conference (NACFC). Although this year is slightly different, considering the…
Recently, gene therapy company Taysha Gene Therapies ("Taysha") announced that its gene therapy candidate, TSHA-102, received both Orphan Drug and Rare Pediatric Disease designations from the FDA. TSHA-102, delivered via…
What are the best treatments and diagnostic approaches for blood disorders like aplastic anemia and myelodysplastic syndromes (MDS)? The UT Southwestern Medical Center is trying to streamline these options and…
According to Newswise, data from a recent clinical trial evaluating IB1001 for patients with Niemann-Pick disease type C (NPC) shows the therapy as effective, safe, and well-tolerated. Sponsored by biopharmaceutical…
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