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RSS FeedPosted by Michael Wonder on 28 Aug 2020
U.S. FDA approves Cystadrops (0.37% cysteamine ophthalmic solution), a new practical treatment option for the ocular manifestations of cystinosis
25 August 2020 - Recordati Rare Diseases today announced the U.S. FDA has approved Cystadrops (0.37% cysteamine ophthalmic solution). ...
Posted by Michael Wonder on 28 Aug 2020
Taysha Gene Therapies receives orphan drug designation and rare paediatric disease designation for TSHA-101 for GM2 gangliosidosis
27 August 2020 - TSHA-101 anticipated to advance into the clinic by the end of 2020. ...
Posted by Michael Wonder on 28 Aug 2020
Rocket Pharmaceuticals receives FDA fast track designation for RP-L401 gene therapy for infantile malignant osteopetrosis
27 August 2020 - Rocket’s fifth gene therapy program receives fast track designation. ...
Posted by Michael Wonder on 27 Aug 2020
COVID-19 vaccines are getting closer. Should we be worried they've come so quickly?
27 August 2020 - Back in March, when the coronavirus pandemic was starting to sweep the globe, a vaccine couldn't ...
Posted by Michael Wonder on 27 Aug 2020
Trump has launched an all-out attack on the FDA. Will its scientific integrity survive?
27 August 2020 - President Trump has instigated an all-out crusade against the FDA at a critical point in the ...
Posted by Michael Wonder on 27 Aug 2020
This is a heart drug that the world doesn’t need
27 August 2020 - Twenty years ago, perhaps the hottest area of drug research was on a class of compounds called ...
Posted by Michael Wonder on 27 Aug 2020
FDA works to evaluate cancer therapies in patients with brain metastases
27 August 2020 - Today, the U.S. FDA issued a draft guidance document, “Evaluating Cancer Drugs in Patients with Central Nervous ...
Posted by Michael Wonder on 27 Aug 2020
Cassiopea receives FDA approval for Winlevi (1% clascoterone cream), first in class topical acne treatment targeting the androgen receptor
27 August 2020 - Cassiopea today announced that the United States FDA approved Winlevi (1% clascoterone cream) for the treatment of ...
Posted by Michael Wonder on 27 Aug 2020
Editas Medicine receives rare paediatric disease designation for EDIT-301 for the treatment of sickle cell disease
24 August 2020 - Editas Medicine today announced that the U.S. FDA has granted rare paediatric disease designation for EDIT-301, an ...
Posted by Michael Wonder on 27 Aug 2020
FDA approves Foundation Medicine's FoundationOne Liquid CDx, a comprehensive pan-tumour liquid biopsy test with multiple companion diagnostic indications for patients with advanced cancer
26 August 2020 - FDA approval includes companion diagnostic claims for Rubraca (rucaparib), the first PARP inhibitor approved in a prostate ...
Posted by Michael Wonder on 26 Aug 2020
Developing safe and effective COVID vaccines — Operation Warp speed’s strategy and approach
26 August 2020 - Announced on May 15, Operation Warp Speed— a partnership of the Department of Health and Human ...
Posted by Michael Wonder on 26 Aug 2020
FDA chief apologises for overstating plasma effect on virus
26 August 2020 - Food and Drug Administration Commissioner Stephen Hahn is apologising for overstating the life-saving benefits of using convalescent ...
Posted by Michael Wonder on 26 Aug 2020
Sarepta Therapeutics announces FDA acceptance of casimersen (SRP-4045) new drug application for patients with Duchenne muscular dystrophy amenable to skipping exon 45
25 August 2020 - Received FDA's conditional approval of Amondys 45 as brand name for casimersen. ...
Posted by Michael Wonder on 26 Aug 2020
Metacrine announces MET409 receives FDA fast track designation for the treatment of NASH
25 August 2020 - Metacrine today announced that the U.S. FDA has granted fast track designation to MET409, the company’s lead ...
Posted by Michael Wonder on 25 Aug 2020
FDA accepts filing of new drug application for tepotinib for the treatment of patients with metastatic NSCLC with METex14 skipping alterations
25 August 2020 - Tepotinib granted priority review and is being evaluated under FDA Real-Time Oncology Review pilot program. ...