Pompe Disease Articles & Analysis: Older
10 news found
It comprises of pluripotent stem cell-derived dopaminergic neurons that are implanted into the brain, thus taking target at the root cause of the disease. According to the Parkinson’s Foundation, more than 10 million people worldwide suffer from Parkinson’s disease. ...
ByBayer AG
“We are excited to combine these unique platforms to develop next-generation genetic therapies with the potential to transform the lives of patients suffering from a wide range of debilitating genetic diseases.” In June 2022, ReCode announced the closing of a $200 million Series B extension financing round, co-led by Leaps by Bayer, the impact investment unit of ...
ByBayer AG
PHILADELPHIA, Oct. 11, 2022 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced four poster presentations highlighting its development program for Pompe disease will be included at the 27th International Hybrid Annual Congress of the World Muscle Society, being held October 11-15, 2022 both virtually and in Halifax, Canada. ...
PHILADELPHIA, Sept. 21, 2022 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced three poster presentations highlighting its development program for Pompe disease will be included at the 2022 American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting, being held September 21-24, 2022 in Nashville, TN and ...
PHILADELPHIA, Aug. 04, 2022 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, today announced financial results for the quarter ended June 30, 2022. ...
(Nasdaq: AVRO), a leading clinical-stage gene therapy company with a shared purpose to free people from a lifetime of genetic disease, today announced preclinical data demonstrating the efficacy and safety of AVR-RD-03 gene therapy in a mouse model of infantile onset Pompe disease. ...
Patient enrollment continues to progress in our GUARD1 Phase 1/2 trial for Gaucher disease type 1, and we look forward to providing a broad program update later in the year that will include new GUARD1 clinical data, as well as outline our development strategy for our Gaucher disease type 3 program. Lastly, we also anticipate regulatory interactions for our ...
We remain deeply committed to bringing AT-GAA to as many people living with Pompe disease as quickly as possible and delivering on our promise to become the potential new standard of ...
Dosed a third patient in our Phase 1/2 GUARD1 clinical trial in Gaucher disease type 1 Deprioritized Fabry disease program in January 2022 to focus pipeline on Gaucher disease programs, cystinosis, Hunter syndrome and Pompe disease programs, extending cash runway into the first quarter of 2024 Presented updated ...
Haymarket Medical Network today launched Rare Disease Advisor, a new digital resource for healthcare professionals (HCPs) to help them recognize, diagnose, refer and treat rare diseases. ...