Real world evidence then becomes even more essential in showing the efficacy of a new treatment as pharma companies try ...

Patient input and experiences play a crucial role in advancing rare disease research and therapy development, as they help define the disease, inform clinical trial design, and influence ...

Roughly one out of every ten people in the United States are affected by rare diseases. Many of these conditions are life‑threatening, and most ...

Ayik is an advocate for people living with rare diseases. Recently, the smoldering embers of conflict in my family’s rare disease community have burst into flames over the use of an eponym ...

Space can be a wondrous place, and we've got the pictures to prove it! Take a look at our favorite space pictures here, and if you're wondering what happened to today in space history don't miss ...

Patient representatives and treatment activists have urged the Government to encourage domestic drugmakers to make generic versions of drugs to treat rare diseases, including spinal muscular ...

(Photo Credit: iStock/Getty Images) The most common ... It's extremely rare, but people with weakened immune systems may develop disseminated disease without treatment. Disseminated disease ...

“Receiving rare pediatric disease designation from the FDA underscores the urgent ... achieve favorable results in clinical trials or preclinical studies or receive regulatory approval on a timely ...

As drugmakers take lessons from oncology in developing CAR T cells for other diseases, they must contend with risks that ...

Panax ginseng shows highest effectiveness in treating Alzheimer's. Learn more about AVXL's blarcamesine and SAVA's simufilam ...

These statements may include the expected timing and results of clinical trials for our product candidates and other clinical and regulatory plans and the market opportunity for those programs. This ...