You're doing the right thing by seeking information, making contacts, and asking questions. With such a rare disease you need to take advantage of the available resources. I have three suggestions for you.
First, learn what you can at these websites, or other trustworthy sites you find:
Second, order the free educational materials
(a set of booklets) on PNH, Clinical Trials, and Bone Marrow and Stem Cell Transplantation from the AAMDS.
Third, find a physician who is a PNH specialist. Since every PNH patient is different, there's both art and science to proper treatment, and not every hematologist knows how to treat PNH. Some names on the PNH Physician List
at the PNH Support Group website may be out-of-date, but you can inquire at local treatment centers or get help from the AAMDSIF (firstname.lastname@example.org or 800-747-2820). If you don't find someone with expertise locally, I'd travel to another city to find the right specialist, at least for a consultation.
Many people live mostly normal lives with PNH. The drug named eculizumab (trade name Soliris) has made a remarkable difference for many patients, and other clinical trials
continue. I have a friend who's had PNH for over 30 years. But PNH can be dangerous, so it can't be ignored.
The information you read online can be comforting or inspirational (see one patient's story
, for example) or can be scary (e.g., dismal survival statistics for some types of bone marrow failure). I've found that information you find online often doesn't represent the current state of the art. For example, current clinical trials or the latest treatment techniques may not yet be reported or reflected in statistics, and older reports based on past results may be just as likely to show up in a search. Working with your physician, you can assess your unique situation.