According to a story from PharmaTimes, the pulmonary arterial hypertension drug selexipag, developed by Actelion, was unable to make the cut to obtain coverage on the NHS in England. The…
Recently in Charcot‐Marie‐Tooth news, progress has been made in identifying better testing to use as outcome measures for people living with Charcot-Marie-Tooth disease type 1A. A study called “Outcome measures…
According to a story from Deseret News, after she was diagnosed with multiple myeloma, Jenny Ahlstrom almost immediately had to make crucial decisions regarding her treatment: decisions that could mean…
Zynerba Pharmaceuticals, Inc. has announced their plans to begin a clinical trial of Cannabidiol in children and teenagers who have Fragile X syndrome. The study, called CONNECT-FX, is expected to…
This is a continuation of An Interview with the Unstoppable Helena Baker. We had just learned about the multiple decades it took for her to receive her diagnosis of Fibula…
According to a July 9 report by the Muscular Dystrophy Association (MDA), the MDA awarded a large grant to researchers studying Charcot-Marie-Tooth disease. The grant, totaling over $1,000,000, was awarded…
The company Pharnext SA, which is developing the investigational drug PXT3003 for the treatment of Charcot-Marie-Tooth disease type 1A, has agreed upon a paediatric investigation plan for the drug with…
The Joshua Frase Foundation has released a new eighteen-minute documentary about the family’s fight for more research into gene therapies for myotubular myopathy, the rare condition that Joshua Frase was…
Danielle and Jonathan Foltz live on the New England coast with their three children. In an CNN article, Wayne Drash documents their meteoric fall from a mission family with big plans…
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.
