Associated tags: Bangladesh Technical Education Board, Boston Children's Hospital, Genetics, Progeria Research Foundation, Hospital, Child, Laminopathy, Family, PRF, NIH, FDA, Disease, Research, Sale, Boston Athletic Association, Pharmaceutical industry, Friends, Emmy Awards, Memory, TED, Attitude, Bank, Syndrome, Athlete, Boston Marathon, Thought, DRS, HBO
Locations: UNITED STATES
Sale,
FDA,
Mortality,
Bankruptcy,
Progeria Research Foundation,
Progeria,
Hearing,
Zydus Lifesciences,
Food,
Lonafarnib,
OTC,
PRF,
HGPS,
Partnership,
Doctor of Philosophy,
Collection,
Research,
European,
Disease,
Patient,
Eiger,
United States bankruptcy court,
Acquisition,
PALO,
DSM-IV codes,
MBA,
Therapy,
Syndrome,
PDPL,
Pharmaceutical industry SOLANA BEACH, Calif. and AHMEDABAD, India and PALO ALTO, Calif., May 3, 2024 /PRNewswire/ -- Sentynl Therapeutics, Inc. (Sentynl), a U.S.-based biopharmaceutical company wholly-owned by Zydus Lifesciences, Ltd. (Zydus Group), and Eiger BioPharmaceuticals, Inc. (OTC: EIGRQ), a commercial-stage biopharmaceutical company focused on the development of innovative therapies for rare metabolic diseases, today announced the closing of the sale of Eiger's Zokinvy® (lonafarnib) program to Sentynl.
Key Points:
- Collectively known as progeria, HGPS and PDPL are ultra-rare, fatal, genetic premature aging diseases that accelerate mortality in young patients.
- Since we first launched PRF in 1999, we have achieved tremendous progress in global awareness, breakthrough research, and treatment of progeria.
- Under the terms of the acquisition, Sentynl acquired global rights to Zokinvy and will be responsible for its manufacture and commercialization.
- For questions on continued access to Zokinvy, please contact the Sentynl Cares support team at 1-888-251-2800 Monday-Friday, 8 am-8 pm ET.
Research,
Friends,
Disease,
Emmy Awards,
Memory,
Partnership,
TED,
Boston Athletic Association,
PRF,
Bank,
Attitude,
Progeria Research Foundation,
Athlete,
Boston Marathon,
Thought,
DRS,
HBO,
FDA,
TED (conference),
Parent,
Boston,
Boylston Street PEABODY, Mass., March 26, 2024 /PRNewswire/ -- The Progeria Research Foundation (PRF), the only nonprofit solely dedicated to finding treatments and the cure for Progeria, celebrates its first year as an Official Charity Partner of the Boston Athletic Association's 128th Bank of America Boston Marathon®. Represented by a team of 10 passionate runners, this partnership will bring vital funds for Progeria research endeavors, while casting a light on this ultra-rare disease and PRF's progress toward the cure.
Key Points:
- PRF's 10 Marathon Runners Raise Funds to cure Progeria, all connected to Sam Berns
PEABODY, Mass., March 26, 2024 /PRNewswire/ -- The Progeria Research Foundation (PRF), the only nonprofit solely dedicated to finding treatments and the cure for Progeria, celebrates its first year as an Official Charity Partner of the Boston Athletic Association's 128th Bank of America Boston Marathon®.
- Without treatment, children with Progeria die of heart disease at an average age of 14.5 years old.
- "From Foxboro neighbors, to middle school classmates, to athletes who got to know Sam at PRF's annual 5K Race for Research – it's heartwarming to know they're now representing The Progeria Research Foundation at the Boston Marathon, in Sam's honor."
- "This is a tremendous honor to be invited to join the Bank of America's Official Charity Program in the 2024 Boston Marathon," said Audrey Gordon, President and Executive Director of The Progeria Research Foundation.
Retrieved on:
Wednesday, January 6, 2021
Senescence,
Progeroid syndromes,
Genodermatoses,
Rare diseases,
Health,
Organic compounds,
Progeria,
Sam Berns,
Lonafarnib,
Berns,
Life According to Sam,
Laminopathy Without the FDA-approved treatment Zokinvy (lonafarnib), children with Progeria die of heart disease at an average age of 14.5 years, due to premature atherosclerosis resulting in heart attacks.
Key Points:
- Without the FDA-approved treatment Zokinvy (lonafarnib), children with Progeria die of heart disease at an average age of 14.5 years, due to premature atherosclerosis resulting in heart attacks.
- Zokinvy increases average lifespan by 2.5 years and improves some symptoms of Progeria, but it does not address the genetic mutation that causes the disease.
- Using a newer form of genetic editing, known as base editing, researchers found that they could reverse the mutation that causes Progeria, improving disease symptoms and dramatically increasing lifespan in a Progeria mouse model.
- The Progeria Research Foundation (PRF) is a nonprofit organization established in 1999 by the family of Sam Berns, a child with Progeria.
Retrieved on:
Friday, November 20, 2020
Branches of biology,
Senescence,
Genodermatoses,
Rare diseases,
Organ systems,
Clinical medicine,
Progeria,
Laminopathy,
HGPS,
HGP,
Hutchinson,
Lamin PRF, a pioneer in the rare disease research foundation space, has led Zokinvy clinical trial research since 2007.
Key Points:
- PRF, a pioneer in the rare disease research foundation space, has led Zokinvy clinical trial research since 2007.
- Without Zokinvy treatment, children with Progeria die of heart disease at an average age of 14.5 years.
- "Progeria is now one of the few rare diseases with an FDA-approved treatment.
- Zokinvy for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and processing-deficient Progeroid Laminopathies is the Company's first FDA approval.
Rare diseases,
Chemistry,
Genodermatoses,
Progeroid syndromes,
Senescence,
Progeria,
Chloroarenes,
Lonafarnib,
Organic chemistry,
Farnesyltransferase,
Progerin,
Biology Children with Progeria live an average 14 years, dying from heart disease typically associated with old age.
Key Points:
- Children with Progeria live an average 14 years, dying from heart disease typically associated with old age.
- Authors from Boston Children's Hospital and Brown University tracked more than 250 children from six continents to demonstrate a link between lonafarnib treatment and extended survival.
- Lonafarnib, originally developed by Merck as a potential cancer therapeutic, inhibits farnesyltransferase, an enzyme that facilitates progerin production.
- In the clinical trial, 27 children with Progeria received oral lonafarnib (150 mg/m2) twice daily as a monotherapy.
