In a recent press release, biopharmaceutical company Akebia Therapeutics ("Akebia") announced the publication of the Phase 3 INNO2VATE study design and methodology. The publication examines the INNO2VATE trial, which evaluated…
Maintaining lung function is a major goal of cystic fibrosis (CF) treatment. In order to accomplish this goal, doctors will use airway clearance techniques, such as vest therapy. The problem…
In a recent press release, the SynGAP Research Fund (SRF) shared that the Heller Laboratory of Neuroepigenetics would receive a $130,000 research grant. The fund, which will be granted over…
In November 2020, Viking Therapeutics ("Viking") shared insight into their metabolic disease program. According to Greg Zante, the Senior VP (SVP) of Finance for Viking, the company's treatment VK2809, licensed…
The 62nd American Society of Hematology (ASH) Annual Meeting and Exposition will be held virtually this year from December 5 through 8. The goal of the meeting is to provide…
COVID-19’s tentacles have invaded the New Drug Applications (NDAs) for 2020. A recent article in Fierce Pharma reported that the FDA has been especially vigilant in scrutinizing NDAs this year,…
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…
This week Hallmark Movies and Mysteries premiered The Christmas Bow, which features Joy Perry, a woman with Charcot-Marie-Tooth disease, playing a woman with CMT. And while CMT has been known…
Innovent Biologics has recently received approval from the National Medical Products Administration (NMPA) for their juvenile idiopathic arthritis (JIA) treatment, SULINNO. It has been approved to treat JIA, rheumatoid arthritis,…
The latest news from Israeli scientists, published in the Times of Israel, is that researchers at Tel Aviv University have accurately targeted cancerous cells in mice using CRISPR.…
On November 19, 2020, Research!America hosted an informational webinar titled "From Nobel Winning Science to Next Generation Treatment: Tracing the Path of a Rare Disease Breakthrough using RNAi." This webinar focused…
According to an announcement from the FH Foundation, data from the FH Foundation’s CASCADE FH Registry, published in The Journal of Pediatrics, has determined that children living with familial hypercholesterolemia regularly…
According Newswise, in a new study first published in Gastroenterology, researchers at Cedars-Sinai have identified a set of genes, made up of rare genetic deviations and more common genetic patterns,…
In a recent press release, gene therapy company Orchard Therapeutics ("Orchard") announced that the FDA approved Orchard's Investigational New Drug (IND) application for OTL-200. This gene therapy solution is…
According to a report in BioPharma Dive, Brainstorm Therapeutics, a New York biotechnology company, said that while its NurOwn therapy for amyotrophic lateral sclerosis (ALS) appeared to have a…
Recently, the American College of Rheumatology (ACR) virtually held ACR Convergence 2020, the organization's annual meeting. At the meeting, researchers shared new insights in the realm of rheumatology; these were…
A recent article in BioSpace highlighted presentations by Twist Bioscience, a rapidly growing genomic and synthetic biology company. Twist presented its product line at the virtual 2020 Annual Meeting of…
As reported in Medical Xpress; Fight for Sight, a charity in the UK dedicated to vision, has an exciting announcement for people with macular diseases: they have just granted funding…
There is a certain time of year in which the trees change color, the air becomes crisp and we pour gravy on everything. As I am typing this, I am…
From November 13-16, researchers, doctors, and others participated in the American Association for the Study of Liver Diseases (AASLD)'s Annual Meeting, or The Liver Meeting Digital Experience. During the…
In late November, RNAi therapeutics company Alnylam Pharmaceuticals ("Alnylam") announced that its therapy OXLUMO (Lumasiran) received approval from the European Commission (EC) for both pediatric and adult patients with…
Thanksgiving is a time of gratitude, family and maybe a little gluttony. It was always my favorite time of the year in high school and college. Now, while I still…
When gene therapy company Asklepios BioPharmaceutical, Inc. ("AskBio") and biotechnology company Selecta Biosciences ("Selecta") joined forces to combat genetic disorders, each company brought its best technology and insights. AskBio…
Colette Wheeler is one of four siblings, and although each had only a 50/50 chance of inheriting the genetic mutation that leads to facioscapulohumeral muscular dystrophy (FSHD), all of them…
Takeda Pharmaceuticals has recently awarded XOMA Corporation $2 million for reaching the next milestone in the development of mezagitamab, a treatment for myasthenia gravis (MG). According to Biospace, XOMA has…
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