Have Homocystinuria and Need Help Affording Your Medical Bills?
Treatment is expensive. The HealthWell Foundation gets it, and they’re here to help. Insurance companies don’t want to pay more than they have to for your medicine. It’s the nature…
Treatment is expensive. The HealthWell Foundation gets it, and they’re here to help. Insurance companies don’t want to pay more than they have to for your medicine. It’s the nature…
Lysogene, a biopharmaceutical company, announced at the end of May that enrollment in SAMOS (Sanfilippo A Multinational Observational Study) was completed. This is extremely exciting news for Sanfilippo syndrome (MPS…
Happy Father's Day Weekend Patient Worthians! This week we have an amazing essay written by CMT Warrior Sarah Magno regarding her personal experience overcoming rare disease obstacles. We also have some…
Want some more news, events and announcements on IPF? We got 'em! [one_half] [/one_half] [one_half_last] Miracle Drug to Become Much More Affordable Soon IPF Treatment Developments You Should…
In late May of this year, Aurinia Pharmaceuticals Inc. dosed its first patient with voclosporin in a Phase 3 trial known as AURORA. AURORA is a confirmatory clinical trial to…
Sarah Trask had childhood cancer 32 years ago and had four brain surgeries. Sarah is a survivor. As a result, epilepsy is the side effect she is living with. Sometimes…
More exciting news from the Annual American Society of Clinical Oncology Meeting last week! Onconova Therapeutics announced results from a Phase 2 study of oral rigosertib as a treatment for…
"You're so full of sh*t." We've all heard it or said it at some point, be honest! But for one 22 year-old man in China, he was literally full of…
In April of this year, Rigel Pharmaceuticals, Inc. announced that it has submitted a New Drug Application (NDA) to the U.S. FDA for fostamatinib, a drug for patients with chronic…
With all the new products, widgets, and gadgets out there meant to clutter our life, there is one that manages to maintain style, grace, and cool points while providing a…
Attention hereditary angioedema patients: preventative treatment is on the horizon! Shire plc is one of the global leaders in rare disease research and pharmacology, and the group recently released positive…
Want some more news, events and announcements on Batten disease? We got 'em! [one_half] [/one_half] [one_half_last] Pioneering Brain Surgery May Give This Toddler More Time FDA Press Release…
GW Pharmaceuticals is finding out... Epidiolex® (manufactured and tested by GW Pharmaceuticals) is a leading medical cannabinoid candidate for the treatment of rare, resistant, and early-onset epilepsy syndromes such as…
Peter Sadler has been raising funds to support the British Lung Foundation, which funds sarcoidosis research. The 42-year-old recently participated in the Bath-Half marathon. He's been on a mission to run in the…
In September, 2015, the U.S. FDA approved NUWIQ®, a recombinant antihemophilic factor (protein essential for normal blood clothing) developed by Octapharma AG, for the treatment of hemophilia A in children…
BrainStorm Cell Therapeutics Inc. is looking to begin producing NurOwn® adult stem cells for their phase III clinical study on ALS patients. The therapeutic company is hoping that the development of…
Though they are frequently misunderstood, genes play an important role in our lives. Most people know that they inherit the majority of their physical features from the genes their parents…
Want some more news, events and announcements on Lennox-Gastaut Syndrome (LGS)? We got 'em! [one_half] [/one_half] [one_half_last] New Research Could Revolutionize LGS and Other Disease Treatments LGS Patients…
Big news for patients with BRCA-mutated metastatic breast cancer: a known ovarian cancer treatment has passed phase III clinical trials for BRCA-mutated metastatic breast cancer, and it's way better than…
The concept of a “library” is something we probably take for granted—but at its core, the library is one of humanity’s greatest innovations. The act of collecting information for the…
Most people diagnosed with ovarian cancer suffer from recurrences after treatment. And every one of them means less and less treatment options. It’s a disheartening, never-ending cycle. What has stumped…
The United States government has certain diseases that they recommend newborn babies to be checked for and currently, spinal muscular atrophy (SMA) isn't on the list. But--great news!--spinal muscular atrophy is…
Want some more news, events and announcements on Fabry disease? We got 'em! [one_half] [/one_half] [one_half_last] This Fabry Disease Treatment Has Had a Rocky Journey Regulatory Pathway Announced…
Welcome to Friday Patient Worthians! This week we have stories on cystinosis and super moms with sickle cell! Additionally, we have a patient story on dystonia and an upcoming event. Lastly, an…
New information about rare diseases is always being discovered. As new symptoms are documented from patients and new research is developed, there’s a good amount of data that needs to…