Patient Advocacy
The United Mitochondrial Disease Foundation is headquarted in this city.
Pittsburgh, PA
LHON
What is Leber's Hereditary Optic Nephropathy?
More than ____% of rare diseases are without an FDA approved treatment.
What is 90%?
Three phosphates and an adenosine.
What is ATP or (adenosine triphosphate)?
This street in Newton was the original location of the Stealth offices.
What is Centre Street?
The recently launched website brings together the mitochondrial disease community through patient stories, articles and videos.
What is TheCharge.com
ADS
What are American Depository Shares?
This organization's slogan is "Alone we are rare, together we are strong".
What is the National Organization for Rare Disorders (NORD)?
There are this many complexes in the electron transport chain? (including atp pump)
What is five?
Who is the longest working employee at Stealth?
Who is Henry Hess?
Both BSF and UMDF put on these types of meetings to help ensure the patients' experiences, perspectives, and needs are captured and incorporated into drug development and evaluation
What is a Patient Focused Drug Development meeting (PFDD)?
The acronym MCSL stands for this.
What is managed care science liaison?
This date is internationally known as Rare Disease Day. It is the "rarest" day of the year.
What is February 28th? (February 29th on leap years)
Each nucleotide is made up of the following three components:
What is a nitrogenous base, a pentose (five-carbon) sugar called ribose, and a phosphate group.
On this date Stealth BioTherapeutics went public?
What is February 14th, 2019?
These two Stealth employees ran the Chicago marathon to raise awareness for mitochondrial disease.
Who are Kayleigh and Reenie?
HIPPA
This law was passed in 1983 to help facilitate the development of rare diseases?
What is the Orphan Drug Act?
This is the balanced scientific equation for cellular aerobic respiration.
C6H12O6 + 6 O2 → 6 CO2 + 6 H2O + energy(ATP)
This higher education school, is named after Gerald Chan's father
What is the Harvard T.H. Chan School of Public Health?
The Mito Foundation, is based out of which country?
What is Australia?
PDUFA
What is the Prescription Drug User Fee Act?
In the United States, a condition is considered “rare” it affects fewer than _________ persons.
What is 200,000 persons?
Taking place in the mitochondrial matrix, this process produces the electrons that travel from one protein complex to another in the inner membrane.
What is the Krebs/citric acid cycle?
At this university, the compound elamipretide was invented.
What is Cornell University?