Neurogene, which works to provide treatments for rare neurological disorders, has recently joined with Behind the Seizure. Behind the Seizure was established by two other companies, BioMarin and Invitae, with…
Congestive heart failure (CHF) is something that affects about six million Americans, and it currently has no cure. Any treatment that does exist for this condition is supportive and symptomatic.…
The Patient Seminar Series is an event in which those who are affected by rare diseases in some way share their experiences. Patients, family members, medical professionals, and legislators all…
NeuroSense Therapeutics, a biotechnology company, is in the process of developing PrimeC. PrimeC was created for the treatment of amyotrophic lateral sclerosis (ALS), and it is meant to slow or…
NeuroQWERTY is a device created by nQ Medical that is meant to monitor brain health and the progression of the disease in those with Parkinson's. It has recently been granted…
Losmapimod is an experimental treatment for facioscapulohumeral muscular dystrophy (FSHD), and it recently received the Orphan Drug designation from the FDA. Not only did it receive this designation, but its…
Front Line Genomics has recently interviewed Dr. Michelle Krishnan, who is the Translational Medicine Leader in Rare Diseases at Roche. She focuses on rare neurodevelopmental disorders, in which she works…
VistaGen Therapeutics has recently been cleared by the FDA to begin the second phase of their study of AV-101, which is intended for the treatment of dyskinesia in those affected…
Dr. Halima Moncreiffe is an assistant professor at Cincinnati Children's Hospital Medical Center, and she focuses on autoimmune diseases. She recently participated in an interview with Front Line Genomics, in…
Zynerba Pharmaceuticals has recently announced that they have finished the patient screening for their clinical trial of Zygel, a treatment for Fragile X syndrome. Zygel is a CBD gel that…
Asfotase alfa is an enzyme replacement therapy meant to directly address the cause of hypophosphatasia. While more research is necessary, it has been shown to decrease the mortality rate and…
PTC Therapeutics is a pharmaceutical company that has been working with Translarna, a medication intended for the treatment of Duchenne muscular dystrophy (DMD). They analyzed this treatment and presented the…
Orchard Therapeutics is investigating their gene therapy, OTL-102, for the treatment of X-linked chronic granulomatous disease (X-CGD). This treatment has recently received the Orphan Drug designation from the FDA, which…
Rubius Therapeutics is a biopharmaceutical company that focuses on cellular medicines. They have recently dosed the first patient in Phase 1b of their clinical trial of RTX-134. This therapy is…
Many diseases are caused by mutations in the DNA. In order to understand these diseases, it is necessary to understand the human genome. Janssen Research & Development has been working…
Over 200,000 people in the United States are affected by acute kidney injury (AKI) every year, with an additional 13 million people across the world also experiencing it. Acute kidney…
When Lily Wythe, a 14-year-old from Eastwood in England, was diagnosed with diffuse pontine glioma (also known as DIPG), her friends and family knew they had to do something to…
Young-onset Parkinson's disease is a mystery to medical professionals. The onset of this condition typically occurs in those older than 60, but in this form the onset occurs between age…
Seelos Therapeutics has won a meeting with the European Medicines Agency (EMA) to advance its therapy, trehalose, for the treatment of neuro-degenerative diseases, specifically Sanfilippo syndrome. At this meeting, which…
A correct diagnosis is often difficult to obtain, especially for a rare disease. Symptoms can be attributed to more common conditions, or they can be written off entirely. Rylee…
Being a teenager is difficult. There are plenty of difficulties and awkward things that teens deal with every day. For those who have a rheumatic disease, their lives can…
About three million Americans are affected by inflammatory bowel disease. Over the years there have been many improvements to treatment for this condition, but there are still further advances that…
AI Therapeutics is a biotechnology company that uses artificial intelligence to match drugs to indications. They do so through the use of their algorithm, Guardian Angel. This algorithm has…
Cancer patients often face adverse effects from their treatment. These effects typically need to be addressed by a doctor, whether they're nausea, rashes, or anything else. The issue is that…
In the wake of Martin Luther King Jr. Day, those with rare diseases have vocalized some of the dreams that they have for the healthcare industry. With all of the…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
