by Peter Ciszewski | Apr 8, 2020
Barbara Burton, MD of Lurie Children’s Hospital of Chicago states why patient registries are important, especially for rare disease populations. As Dr. Burton explains there are several reasons why patient registries are important, including the value they...
by Peter Ciszewski | Apr 7, 2020
CheckRare is working to keep our audience of health care professionals up-to-date on the latest guidances and developments as it relates to how to better manage rare disease patients, many that require special medications and special care, during the current COVID-19...
by Peter Ciszewski | Apr 6, 2020
Necrotising fasciitis (NF), commonly known as flesh-eating disease, is an infection that results in the death of the body’s soft tissue. It is a severe disease of sudden onset that spreads rapidly. Symptoms include red or purple skin in the affected area, severe...
by Peter Ciszewski | Apr 6, 2020
Melissa Wasserstein, MD, from the Children’s Hospital at Montefiore, talks about Acid Sphingomyelinase Deficiency (ASMD), also known as Niemann-Pick Diseases type A or type B. ASMD is a genetic disorder caused by mutations in the SMPD1 gene that encodes for ...
by Peter Ciszewski | Apr 2, 2020
Geoff MacKay, president and CEO of Avrobio, described the company’s history and philosophy for developing ex vivo gene therapies for various diseases, such as Pompe disease, Fabry disease, Gaucher disease, and cystinosis. As Mackay explains in this video, the...
