Associated tags: LAMP2, Danon disease, Patient, Safety, Clinical Trials, RP-A501, Health, Male, Pharmaceutical industry, Medication, Pharmaceutical, Cardiac muscle, Disease, Tissue, LVV, Medical device, Pyruvate kinase deficiency, Glycogen, Autophagosome, FDA
Retrieved on:
Tuesday, September 12, 2023
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LAMP2 The disease affects an estimated 15,000 to 30,000 patients in the U.S. and Europe.
Key Points:
- The disease affects an estimated 15,000 to 30,000 patients in the U.S. and Europe.
- A global natural history study will serve as an external comparator and run concurrently to the Phase 2 pivotal trial.
- In-house manufacturing has been completed with sufficient high-quality drug product produced to fully supply the Phase 2 pivotal study.
- Additionally, Rocket has secured an ICD-10 code from CMS for LAMP2 deficiency in Danon Disease
Retrieved on:
Friday, September 30, 2022
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NYHA Further, efficacy data from the pediatric patients are following similar or more favorable positive trends as in the adults at a similar timeframe.
Key Points:
- Further, efficacy data from the pediatric patients are following similar or more favorable positive trends as in the adults at a similar timeframe.
- RP-A501 is an investigational gene therapy product being developed for Danon Disease and the first potential gene therapy for monogenic heart failure.
- Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition.
- Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes.
Retrieved on:
Monday, November 15, 2021
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DANON DISEASE,
ROCKET PHARMACEUTICALS, INC. Male patients living with Danon Disease suffer a heavy disease burden and face rapidly progressive heart failure in their teenage years.
Key Points:
- Male patients living with Danon Disease suffer a heavy disease burden and face rapidly progressive heart failure in their teenage years.
- The clinical data to date also demonstrate encouraging results including clinical stabilization of this rapidly progressive disease.
- Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition.
- Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes.
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DANON DISEASE,
ROCKET PHARMACEUTICALS, INC. Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that updated data from the ongoing Phase 1 clinical trial of RP-A501 in Danon Disease will be presented at the American Heart Association (AHA) Scientific Sessions 2021 taking place virtually November 13-15, 2021.
Key Points:
- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that updated data from the ongoing Phase 1 clinical trial of RP-A501 in Danon Disease will be presented at the American Heart Association (AHA) Scientific Sessions 2021 taking place virtually November 13-15, 2021.
- RP-A501 is an investigational gene therapy product being developed for Danon Disease and the first potential gene therapy for monogenic heart failure.
- Danon Disease is a rare X-linked inherited disorder caused by mutations in the gene encoding lysosome-associated membrane protein 2 (LAMP-2), an important mediator of autophagy.
- Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition.
Retrieved on:
Tuesday, September 14, 2021
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ROCKET PHARMACEUTICALS, INC. Steroid-induced myopathy was observed in two of the three patients >2 weeks after dosing, which also resolved.
Key Points:
- Steroid-induced myopathy was observed in two of the three patients >2 weeks after dosing, which also resolved.
- In these two patients, a substantial improvement of a key marker of heart failure, B-type natriuretic peptide (BNP), was also observed.
- RP-A501 is an investigational gene therapy product being developed for Danon Disease and the first potential gene therapy for monogenic heart failure.
- Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition.
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Rocket Pharmaceuticals, Inc.,
RP-A501,
DANON DISEASE,
ROCKET PHARMACEUTICALS, INC. We are grateful for the collaboration between the FDA and our team in reaching agreement on protocol updates allowing us to resume patient enrollment in our Danon Disease trial.
Key Points:
- We are grateful for the collaboration between the FDA and our team in reaching agreement on protocol updates allowing us to resume patient enrollment in our Danon Disease trial.
- We look forward to progressing this critical work on behalf of all Danon patients, said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma.
- Rockets Danon Disease program was placed on clinical hold by the FDA in May of 2021 to modify the study protocol and other supporting documents with revised guidelines for patient selection and safety management.
- Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition.