Krista Rossi

The phase 1/2 trial for ABO-102 (AAV-SGSH), clinical gene therapy for the treatment of Sanfilippo syndrome type A (MPS III A) shows efficacy in trial update.

The FDA has granted Rare Pediatric Disease Designation to Myonexus Therapeutics for its MYO-101, which is an AAV-based gene therapy for the treatment of limb girdle muscular dystrophy (LGMD) type 2E.

The FDA granted rare pediatric disease designation to Castle Creek Pharmaceuticals (CCP) for diacerein 1% ointment (CCP-020) for the treatment of epidermolysis bullosa simplex (EBS).

The US Food and Drug Administration (FDA) has accepted the Bioverativ's Investigational New Drug (IND) application for BIVV003, a gene-edited cell therapy candidate for the treatment of people with sickle cell disease.

Data is released from a pilot study of X4P-001-IO in combination with nivolumab in patients with clear cell renal cell carcinoma (ccRCC) who are non-responsive to the anti-PD-1 checkpoint inhibitor nivolumab alone.

FDA grants orphan drug designation to Sarconeos for Duchenne muscular dystrophy (DMD).

Positive results yield from a phase 2 tudy of bertilimumab in patients with moderate-to-extensive bullous pemphigoid (BP).

FDA approves Retacrit (epoetin alfa-epbx) as a biosimilar to Epogen/Procrit (epoetin alfa) for the treatment of anemia caused by chronic kidney disease, chemotherapy, or the use of zidovudine in patients with HIV infection.

The first patient has been dosed in aphase 1/2 study (BMN 270-203) evaluating the investigational gene therapy, valoctocogene roxaparvovec, in severe hemophilia A patients with pre-existing AAV5 antibodies.

FDA Approves SC Formulation of Tocilizumab for Polyarticular Juvenile Idiopathic Arthritis (PJIA).

LogicBio Therapeutics Inc will present data regarding gene editing technologies in hemophilia B and Methylmalonic Acidemia (MMA) at the annual meeting of the ASGCT in Chicago.

AMO Pharma Limited announces the commencement of patient recruitment for an interventional study of AMO-01, an investigational Ras-ERK pathway inhibitor for the treatment of Phelan-McDermid syndrome (PMS).

On May 17th, the National Organization of Rare Disorders (NORD) will host its 2018 Rare Impact Awards in Washington, DC at the Andrew W. Mellon Auditorium.

The first patient has been dosed in the Phase 1/2 clinical trial of its drug, KB103, for the treatment of dystrophic epidermolysis bullosa (DEB).

Study show blocking FOXM1 can reduce the activation of fibroblasts as well as the process of idiopathic pulmonary fibrosis (IPF) itself.

Results show that blade plates or intramedullary nails placed via surgery can be effective treatments for patients with fibrous dysplasia of the proximal femur.

The US FDA has granted Palladio Biosciences Investigational New Drug (IND) clearance to proceed with a Phase 2 clinical trial of lixivaptan capsules in patients with autosomal dominant polycystic kidney disease (ADPKD).

DNAtrix will report data from an ongoing Phase 1 trial of DNX-2401 in pediatric patients with newly diagnosed diffuse intrinsic pontine gliomas (DIPG), which indicated efficacy through a prolonged survival rate.

Results from the phase 3 FACETS trial showed that migalastat improved diarrhea in patients with Fabry disease.

In the recent review, it was shown that surgery can cure nearly all patients with the focal or localized form of congenital hyperinsulinism and prevent complications in patients with the diffuse from in which insulin-producing cells occur throughout the pancreas.

Bruce Wang, MD, a porphyrias specialist and assistant professor in the gastroenterology division in the department of medicine at University of California San Francisco (UCSF) and UCSF Porphyria Center and the Porphyrias Consortium, explains acute hepatic porphyrias (AHPs).

FDA approves dual drug treatment, dabrafenib (Tafinlar) and trametinib (Mekinist), for Anaplastic Thyroid Cancer (ATC).

Cytokinetics, Incorporated and The ALS Association continue their partnership in an effort to fight ALS.

Lyso-Gb1 Identified as most efefctive biomarker for Gaucher disease monitoring.

Clinical and biochemical assessments of LPL FCS and non-LPL FCS genes.

FCS community highlights clinical presentation and lifestyle.

Treatment with volanesorsen shows efficacy in patients with FCS.

Dystrophic epidermolysis bullosa treatment receives IND clearance.

Data from Type 1 spinal muscular atrophy (SMA) treatment suggests efficacy and tolerability.

CLN2 disease treatment reduces rate of clinical decline in children.