Enzyme Images Help Solve Cause of Rare Neurodegenerative Diseases
According to a recent article, researchers were able to produce the first images of an enzyme that may figure out the unknown cause of a
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JumpStart
JumpStart
The Orphan Disease Center’s JumpStart program serves to establish and progress research agendas in emerging and neglected rare diseases. The JumpStart program partners with patient groups and families to address gaps by identifying key opinion leaders and introducing new researchers to a disease. The program can help facilitate the development of animal models, establish a patient alliance or a patient registry, and organize symposia. Due to limited resources, ODC is unable to provide financial support. JumpStart primarily offers scientific advisement and network outreach.
Condition Awareness & Advocacy
Here is a list of conditions this partner raises awareness and advocacy for:
- Emerging and neglected rare diseases
Patient Worthy Posts on Rare Disease
Doing What is Best For the Patient May Override a Doctor’s Compassion
Compassion [kuhm–pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the
Study: Opportunities and Challenges for Newborn Screening
Newborn screening (NBS) is a public health service within the United States through which babies are screened for medical conditions that otherwise wouldn’t be apparent
Science Simplified: What is a Natural History Study?
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works
Compassion Corner: Compassionate Use, Expanded Access Part Two: The Terminology Is Different But the Unmet Need Is the Same
Continued From Part One Compassion [kuhm–pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong
ADVM-062 for BCM Earns Orphan Drug Designation
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases. For the purpose
