Respiratory disorders can be common, but for more information on rare conditions like Cystic Fibrosis, make Rare Disease Report your primary resource.
How to Manage an NTM Infection
Most people do not become ill from Nontuberculous mycobacteria (NTM), but in people of compromised health, an infection can lead to more serious problems.
Interim data from an ongoing, open-label, pilot Phase 2 study of XmAb5871 in patients with IgG4-Related Disease (IgG4-RD) was presented today at EULAR 2017.
Nucala (mepolizumab) helped about one-half of the patients with refractory or relapsed eosinophilic granulomatosis with polyangiitis – a patient population that has been historically difficult to treat.
The Boomer Esiason Foundation (BEF) has recognized 2 cystic fibrosis teens as recipients of its annual “Team Boomer Athlete of the Year” award.
By having all carriers of a monogenic rare disease undergo the IVF procedure to create children, that rare disease could be eradicated.
June 2017 has been designated as the first-ever Worldwide LAM Awareness Month to educate all people from all countries about lymphangioleiomyomatosis.
Janet Woodcock: “Many rare cystic fibrosis mutations have such small patient populations that clinical trial studies are not feasible"
The Mauli Ola Foundation (MOF) are a team surfers who help cystic fibrosis patients breathe better. And it is based on solid scientific evidence.
Born without a lower jaw, Isaiah can’t breathe through his mouth and he has never spoken a word. In spite of it, the 17-year-old released his first rap song.
BPompe disease is a rare lysosomal storage disorder that impacts muscle strength. Weak muscles of the airways and tongue can affect patients' ability to talk, breath, and swallow.
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