Our growing community is essential to catalyze research and development of life-saving therapeutics. Also essential is the perspective that each of our community members can offer the broader rare disease community. In 2021, our community helped to spread awareness for our cause and amplify their voices through our blogs.

We are proud to share our top blogs from 2021 with you here:

POTS Awareness - Katie Kuhl
  1. Postural Orthostatic Tachycardia Syndrome (POTS)

CRD intern Katie Kuhl shares her daily life with Postural Orthostatic Tachycardia Syndrome on POTS Awareness Day. 

What they don't tell you about Duchenne
  1. What They Don't Tell You About Duchenne

Having a loved one with rare disease comes with a whole host of challenges that no one can be prepared for. Dawn Jones shares what it’s like to be strong for her son with Duchenne, while also facing the crushing weight of the reality of Duchenne.  

A Timeline of the Science Behind Cure Rare Disease
  1. A Timeline Of The Science Behind Cure Rare Disease

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 more about the history behind the technology being used in our custom therapeutics. 

Findings From CRD's AAV Seropositivity Study
  1. Findings From Cure Rare Disease's AAV Seropositivity Study

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 of our NAbs testing program, we share the results so far and what they mean for patients and the future of gene therapy. 

My Child in Disguise
  1. My Child in Disguise

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 Joaquin change as a result of these steroids. 

Cure Rare Disease 3 Years Later
  1. Cure Rare Disease - 3 Years Later

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. 

Building a New Future
  1. Building a New Future: Our Family’s Floor Plans to a New Accessible Home

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 sons with Duchenne. 

Elijah Stacy, author with Duchenne
  1. Q&A with Elijah Stacy, author of “A Small If,” a memoir on living with Duchenne muscular dystrophy

In this conversation with Elijah Stacy, an author with Duchenne, he shares the story behind his memoir and his hopes for the future. 

The Precious Gift of a Rare Disease Diagnosis
  1. The Precious Gift of a Rare Disease Diagnosis

Receiving a rare disease diagnosis can be hard to come to terms with, but it is important to keep the small blessings in mind. Kati Falger talks about how her son’s DMD diagnosis has changed her perspective and daily life in general. 

Justin Cohen
  1. Overcoming Obstacles: Building a Successful Career in an Inaccessible World

In a world that was not designed for those with physical disabilities, it can be difficult to succeed when all the odds seem to be stacked against you. Justin Cohen shares his rare disease story and his journey in getting to the career he has now as a post-doctorate in the Lek Lab.