Krista Rossi

Dystrophic epidermolysis bullosa (DEB) treatment granted OMPD in Europe.

The first patient was dosed in X-linked retinitis pigmentosa (XLRP) treatment.

This morning, the FDA granted Orphan Drug Designation to KL1333 for the treatment of inherited mitochondrial respiratory chain diseases (MRCD).

FDA grants Breakthrough Therapy Designation to emicizumab-kxwh (Hemlibra) for people with hemophilia A without factor VIII inhibitors.

Can the Epstein-Barr virus cause autoimmune illnesses?

FDA approves the development of VAR 200 for the treatment of focal segmental glomerulosclerosis (FSGS) in adults.

FDA exercises enforcement discretion regarding its ExeGen ATM MiniSwine model, clearing the genetically engineered (GE) to model ataxia telangiectasia (AT) for commercial use as a research tool.

Positive pre-clinical data is released regarding BGB601 for the treatment of fibrosis, idiopathic pulmonary fibrosis (IPF), and non-alcoholic steatohepatitis (NASH).

FDA approves Phase 2 study of KD025, a a possible treatment for graft-versus-host disease (GVHD).

CEO of Krystal Biotech discusses first topical gene therapy for dystrophic epidermolysis bullosa.

The self-administration of pnf C1-INH (Berinert) proves effective for patients with hereditary angioedema with C1 inhibitor deficiency.

Higher-quality epidemiological data is needed in all 4 subtypes of Sanfilippo syndrome for better disease research and management.

In a recent interview with Dr Steven Freedman, Rare Disease Report asked for the specifics on familial chlyomicronemia syndrome (FCS).

FDA grants orphan drug designation to elamipretide for the treatment of Barth syndrome.

Novartis and AveXis merge spells opportunity for SMA research.

Rhizen Pharmaceuticals SA's product, Tenalisib (RP6530), is granted orphan drug designation from the FDA for the treatment of CTCL.

QurAlis Corporation joins forces with investors MP Healthcare Venture Management (MPH), Amgen Ventures, and Alexandria Venture Investments to find a cure for ALS.

Phase 2 Study with Lanifibranor on Non-Alcoholic Fatty Liver Disease in Patients with Type 2 Diabetes Commences.

The FDA granted orphan drug designation to fluasterone (ST-002) for the treatment of Cushing’s syndrome.

The FDA has accepted BLA for hairy cell leukemia treatment.

Altemia, a new treatment for sickle cell disease (SCD), was granted orphan drug designation.

REVERSE Phase III clinical trial shows efficacy and safety of GS010 in patients with LHON.

Phase 3 OLYMPUS clinical trial releases interim analysis of UGN-101 (MitoGel) for low-grade UTUC.

FDA grants orphan drug designation to PharmAbcine Inc for TTAC-0001, the company’s leading clinical compound, for treatment of GBM.

Catalyst Pharmaceuticals announces its submission of an NDA to the FDA for amifampridine phosphate (Firdapse) for the treatment of LEMS.

The FDA granted orphan drug designation to Scholar Rock for its lead antibody product candidate, SRK-015, for the treatment of spinal muscular strophy (SMA).

Phase 3 of the NEWTON 2 study of EG-1962 in aSAH has been terminated since it is unlikely to meet its primary endpoint.

The FDA granted Cynata’s Therapuetics Limited (ASX:CYP) orphan-drug designation for CYP-001, which is the company’s potential treatment for acute graft versus host disease (GvHD).

The FDA granted GeneTx Biotherapeutics LLC orphan-drug designation for GTX-101, the company’s potential treatment for Angelman syndrome.

Eiger announced first patient treated in the PREVENT study, a phase 2, multicenter study of subcutaneous (SC) exendin 9-39 in post-bariatric surgical patients who experience PBH.