RDR Alert®

Last night, SELLAS Life Sciences Group, Inc. announced that the U.S. FDA has granted orphan drug designation to galinpepimut-S (GPS) for the treatment of multiple myeloma.

Cellectar Biosciences has announced that the U.S. FDA granted orphan drug designation to CLR 131 for the treatment of rhabdomyosarcoma, a rare pediatric cancer.

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

New data revealed by the Tufts Center for the Study of Drug Development concludes that orphan drug development takes 18% longer than the average time required for other new drugs.

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

Rare Disease Report sat down with AdAlta CEO Sam Cobb to discuss the impetus for the redesign of AD-214, and what it means for those in the idopathic pulmonary fibrosis.

Biohaven Pharmaceutical has established an expanded access program (EAP) with sublingual BHV-0223 for patients with amyotrophic lateral sclerosis (ALS).

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.

Alnylam Pharmaceuticals has achieved delivery of novel small interfering RNA (siRNA) conjugates to the central nervous system and intends to advance a pipeline of investigational RNAi therapeutics into clinical development.

The FDA has approved daratumumab (Darzalex) in combination with bortezomib (Velcade), melphalan, and prednisone (VMP) for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant (ASCT).

Wahida Karmally, MS, RD, CDE, explains the challenges and importance of diet in patients with familial chylomicronemia syndrome (FCS), how much fat can be eaten (and which kind), and the dangers of pancreatitis when too much fat is consumed.

Results from the pivotal QuANTUM-R phase 3 study of single agent quizartinib significantly prolongs overall survival compared to chemotherapy in patients with relapsed/refractory AML with FLT3-ITD mutations.

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.

A human clinical trial grant totaling $750,000 was awarded this morning by the Muscular Dystrophy Association (MDA) to Massachusetts General Hospital, with the hope that it will accelerate biomarker research for amyotrophic lateral sclerosis (ALS).

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).

In an interview with Rare Disease Report, Mathew Maurer, MD, Arnold and Arlene Professor of Cardiology at Columbia University, explained the standard of care and current management tactics for individuals with hATTR Amyloidosis.

Abhimanyu Garg, MD, discusses the cofactors and concerns in FCS treatments.

Abhimanyu Garg, MD, indicates the importance of neonatal screening for familial chylomicronemia syndrome (FCS).

This morning, it was announced that Invitae Corporation and Sarepta Therapeutics will be expanding its partnership to continue assisting clinicians in identifying patients with Duchenne muscular dystrophy (DMD).

The U.S. FDA has granted Fast Track designation to PaxVax for development of its vaccine for prevention of the chkungunya virus.

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

Rare Disease Report sat down with Brian Wamhoff, PhD, Head of Innovation at HemoShear Therapeutics, to discuss what the company is doing throughout the rare disease community and why it could have such an incredible impact in the near future.

Alan S. Brown, MD, FNLA, FACC, FAHA is a Cardiologist at Advocate Lutheran General Hospital and the President-Elect of the National Lipid Association (NLA). While at the NLA’s 2018 Scientific Sessions in Las Vegas, Nevada, he explained the connection between hypertriglyceridemia and FCS.