As mobility decreases, typical houses may become inaccessible for people with Duchenne. JeanAnn Bryan shares how she and her family built a new home that was accessible for her two […]
In the three years since the founding of Cure Rare Disease, there have been many accomplishments on our journey to creating the first custom drug therapy for rare disease patients. […]
Though steroids can slow the progression of Duchenne, they come with a variety of difficult side effects. Sara Aluffi talks about the varying emotions that come with watching her son […]
In 2020, we launched our study into our patient’s AAV neutralizing antibodies, a key factor in how effective certain gene therapies may be for them. A year after the start […]
Cure Rare Disease would not be where it is today without the work of previous scientists in developing CRISPR technology. As we approach the dosing of our first patient, learn […]
CRD intern Katie Kuhl shares her daily life with Postural Orthostatic Tachycardia Syndrome on POTS Awareness Day.