In a recent press release, biopharmaceutical company Fortress Biotech announced that their study regarding ATP7A targeted next generation DNA sequencing for Menkes disease was published in Molecular Genetics and Metabolism…
Full Patient Enrollment in Study Expected by Year-End 2019 LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Feb. 04, 2019 -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for…
CHICAGO, July 19, 2018 – The Pulmonary Fibrosis Foundation (PFF) and monARC Bionetworks have partnered to create the mobile app, PF Health, for individuals living with pulmonary fibrosis (PF). PF Health, now available on Apple App Store (iOS 9.0 or…
BOHEMIA, N.Y., June 25, 2018 (LGS Foundation) – The LGS Foundation is pleased to announce that the U.S. Food and Drug Administration (FDA) has approved EPIDIOLEX® (cannabidiol / CBD) for the…
NEW YORK (PRWEB) MARCH 13, 2018 – The American Liver Foundation (ALF), the nation’s largest patient advocacy organization for people with liver disease, announced today the launch of an online…
Press Release: Lend Your Voice to the Fight to Breathe Faced by the UK’s Cystic Fibrosis Community Cystic fibrosis families from around the UK have joined forces to release a…
Date of issue: October 19, 2017 Boxing legend Michael Carruth was today firmly in Cystic Fibrosis Ireland's corner at Dublin's Irish Film Institute for the launch of a new awareness…
It’s always exciting when two giants of their fields join together to accomplish something. When Pixar and Disney joined forces to bring us the best animated films since… ever, the…
Press release: 12th October 2016 Abigail Brundrett, 16 and Cassidy Vancil, 18 from Charlotte, NC, have choreographed and performed a dance video [to help her lifelong friends]. [It] highlights the struggle faced by people…
Press release: 6th September 2016 Raise awareness about your cystic fibrosis issues on Worldwide CF Day Since 2013 many countries have marked Worldwide CF Day on the 8th September. As this…
¡NOTICIAS DE ÚLTIMA HORA! Pronto puede haber otro tratamiento para pacientes del IP! La compañía ADMA Biologics, Inc. ha desarrollado una nueva opción de tratamiento, RI-002, para los pacientes que…
Press release - 10th July 2016 PW Partner: Breathe With Me, Strawfie Challenge When Danielle Cartwright filmed her daughter Talia May taking her medication and proudly posted the video online,…
Press release: 2nd June 2016 Xenia twins urge local community to take the Strawfie Challenge for cystic fibrosis awareness When Heather Scott and Nathan Young received the diagnosis that their daughter Adriana…
Press release: 24th May 2016 Breathe With Me Strawfie Challenge The Team GB Trampoline Squad are the latest in a fast-growing line of sporting stars and celebrities to back the ‘Breathe…
You may have seen the Jeffery Modell Foundation's name around town, literally, from their educational posters. I was thrilled to come across one in the Chicago O'Hare International Airport. The…
Post Rare Disease Day 2016 the FDA's Office of Special Medical Programs announced a new program that would work to provide funding to patient advocacy groups and other organizations who were studying…
Study Title: Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of N91115 to Evaluate Efficacy and Safety in Patients With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation Treated With…
CYSTIC FIBROSIS PATIENTS VOTE FOR ALTERNATIVES TO TRADITIONAL TREATMENT CysticLife partners with researchers at Mayo Clinic to study if exercise can replace therapy 1x per day Phoenix, AZ: Fed…
BREAKING NEWS! There may soon be another treatment for PI patients! The company ADMA Biologics, Inc. has developed a new treatment option, RI-002, for patients who are immune-compromised. It’s currently…
© Copyright 2024 Patient Worthy
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.
