RDR Alert®

Dosing of the first patient in the Phase 2 clinical trial of AG10 in patients with ATTR cardiomyopathy, Eidos Therapeutics, Inc. has announced.

This past year, James A. Underberg, MD, MS, FACPM, FACP, FASPC, FNLA, President of the National Lipid Association (NLA), outlines familial chylomicronemia syndrome (FCS) and the need for treatments, which may not be far.

On Monday, the FDA approved tisagenlecleucel (Kymriah) for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma who have relapsed or are ineligible for an autologous stem cell transplant (ASCT).

Orchard Therapeutics announced that its gene therapy candidate, OTL-200, has been granted Rare Pediatric Disease designation for the treatment of metachromatic leukodystrophy.

At the National Lipid Association's (NLA) 2018 Scientific Sessions in LAs Vegas, NV, Wahida Karmally, MS, RD, CDE, explains the importance of nutrition and support in patients with familial chylomicronemia syndrome (FCS) and the emotional stress a strict, regimented diet can have on patients.

Akcea Therapeutics announced that it has expanded its global early access program to the US for its drug volanesorsen, which is in development for treatment of the rare lipid disorder FCS.

REGENXBIO Inc. announced that the U.S. FDA has granted Fast Track designation to RGX-131, a novel, one-time investigational treatment for mucopolysaccharidosis type II (MPS II).

A Rare Pediatric Disease designation was granted by the U.S. FDA this morning to Cellectar Biosciences for CLR 131, intended for the treatment of neuroblastoma.

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.

As stated in article published in the clinical journal Neurobiology of Aging, a team of researchers has discovered that elevated levels of certain types of lipids in the brain could be an early presentation of Parkinson’s disease.

This morning, Acceleron Pharma announced that the US FDA has granted Fast Track designation to ACE-083 for the treatment of patients with facioscapulohumeral muscular dystrophy (FSHD).

This morning, it was announced that the U.S. FDA has approved dabrafenib (Tafinlar) in combination with trametinib (Mekinist) for the adjuvant treatment of patients with melanoma with BRAF V600E or V600K mutation.

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

Positive results from the Fostamatinib in Thrombocytopenia (FIT) Phase 3 clinical program of fostamatinib disodium hexahydrate (Tavalisse) were published in the American Journal of Hematology this week.

FCS community highlights clinical presentation and lifestyle.

At the Clinical immunology Society Annual Meeting in Toronto on April 27-28, Prometic presented data from its pivotal Intravenous Immunoglobin (IVIG) Phase 3 clinical trial.

The U.S. FDA has accepted for priority review the Biologics License Application for cemiplimab (REGN2810), an investigational anti-PD-1 monoclonal antibody for patients with metastatic CSCC.

This morning, Capricor Therapeutics, Inc. announced that the HOPE-2 clinical trial has been initiated at UC Davis Medical Center in Sacramento, CA.

Treatment with volanesorsen shows efficacy in patients with FCS.

Results from a questionnaire show that individuals living with FCS prove that communication among people in similar situations can improve a patient’s quality of life.

At the NLA Scientific Sessions, Akcea Therapeutics presented a poster with data from 2 different studies, each of which exhibited a significant reduction of triglycerides in patients with FCS when administered volanesorsen.

At the NLA Scientific Sessions in Las Vegas this morning, Akcea Therapeutics, presented a poster with data from the largest burden of illness study in patients with FCS to date.

At the NLA Scientific Sessions in Las Vegas, NV this weekend, Akcea Therapeutics will present clinical data on volanesorsen and FCS.

This morning, biotechnological company Saniona announced that it has obtained approval to initiate the second part of its Phase 2a trial for Tesomet in adolescents with Prader-Willi syndrome (PWS).