Drug Discovery World
JUNE 23, 2023
The Food and Drug Administration (FDA) has granted accelerated approval to Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus (AAV) based gene therapy for the treatment of DMD. The FDA’s accelerated approval is based on an increase in Elevidys micro-dystrophin protein expression in skeletal muscle.
Advarra
JANUARY 11, 2024
The applications of mRNA-based therapies in cancer research represent one of the next groundbreaking steps toward improved cancer treatments. Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials.
This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.
The Pharma Data
JANUARY 15, 2021
hereafter, Daiichi Sankyo) and AstraZeneca’s Enhertu ( fam-trastuzumab deruxtecan -nxki) has been approved in the U.S. for the treatment of adult patients with locally advanced or metastatic HER2 positive gastric or gastroesophageal junction (GEJ) adenocarcinoma who have received a prior trastuzumab-based regimen. In the U.S.,
Conversations in Drug Development Trends
MAY 30, 2024
Since marketing authorization for the first breakthrough treatment in 1994, the steady increase in clinical trials reflects the community’s commitment to finding effective ALS treatments despite the numerous hurdles associated with clinical trial design, from proof-of-concept to pivotal trials. Initial approval in 2022: FDA, U.S.
The Pharma Data
SEPTEMBER 28, 2021
additionally , all five babies maintained the power to swallow and were ready to feed exclusively orally after 12 months of treatment. Food and Drug Administration (FDA) and European Medicines Agency (EMA) approved Evrysdi for the treatment of SMA in adults and youngsters 2 months aged and older.
The Pharma Data
DECEMBER 8, 2020
Li stated, “One in every six people in China is living with a CNS condition, yet there are relatively few effective treatments available today, underscoring the urgent need to develop and deliver novel, effective therapies. Pear’s reSET, reSET-O and Somryst are the first PDTs to receive FDA approval for treating disease.
Alta Sciences
APRIL 17, 2024
Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.
Expert insights. Personalized for you.
243 
Let's personalize your content