RDR Alert®

Data from a Phase 2 trial of galinpepimut-S (GPS) in acute myeloid leukemia (AML) have been published in the current issue of the peer-reviewed medical journal Blood Advances.

Thanks to an outpouring of support from the congenital muscular dystrophy (CMD) community over the past year, Cure CMD was able to announce new grant funding for 2018-2019.

Data recently published in the Orphanet Journal of Rare Diseases showed that long-term improvements and stability can be achieved with taliglucerase alfa in patients with Gaucher disease.

George Yohrling, Ph.D., Senior Director of Mission and Science at the HDSA discusses the challenges that come with working with patients with HD, and communicating with their family members and caregivers.

15-year old biliary atresia patient Jarrius Robertson will be honored by the WWE. At the 2018 WWE Hall of Fame Induction Ceremony on April 6, he will become the fourth recipient of the Warrior Award.

On Rare Disease Day 2018, Shire plc will focus on promoting prompt diagnosis of rare diseases, as it is the most important issue affecting health, longevity and well-being for patients and their families..

Positive outcomes were reported from a biomarker study evaluating intracellular glycosaminoglycans (GAGs) levels in leukocytes as a disease activity biomarker in MPS VI.

The National Center for Advancing Translational Sciences and the National Institutes of Health Clinical Center will honor World Rare Disease Day this year by hosting an event to celebrate patient advocacy.

Last week in Nature, data were published revealing that AD-114, a locally developed molecule, could be a therapeutic option for patients with idiopathic pulmonary fibrosis.

Fortress Biotech and its subsidiary Aevitas Therapeutics have entered into a sponsored research agreement with Dr. Guanping Gao’s laboratory at the University of Massachusetts Medical School.

This morning, Children’s National Health System and Horizon Pharma plc have announced the creation of the Horizon Pharma Clinical Care Endowment at the Children’s National Rare Disease Institute.

On Rare Disease Day, a tribute committee will rename a plaza in Cambridge, Massachusetts in honor honor of Henri A. Termeer, late biotechnology pioneer and former Genzyme Chief Executive Officer.

John Crowley, the President and CEO of Amicus Therapeutics, sat down with Rare Disease Report at the 14th Annual WORLDSymposium in San Diego to discuss the President's mention of rare diseases.

The Committee for Medicinal Products for Human Use of the EMA recommended that both Mylotarg (gemtuzumab ozogamicin) and Bosulif (bosutinib) be reviewed separately by the European Commission.

Sobi announced that the EMA's Committee for Medicinal Products for Human Use has adopted a positive opinion for anakinra (Kineret) for the treatment of Still’s disease.

Kazia Therapeutics has received orphan drug designation by the U.S. FDA for GDC-0084, intended for the treatment of glioblastoma multiforme.

At the Cost-Sharing Roundtable on Friday, K. Robin Yabroff, Ph.D. presented data explaining the economic struggles that can be had by those who need medical attention.

After WORLDSymposium, Rare Disease Report spoke with Loren D.M. Pena, M.D., Ph.D., who presented new safety data for neoGAA in late-onset Pompe.

Vertex has initiated a Phase 3 study of VX-659, Tezacaftor and Ivacaftor for people with cystic fibrosis who have one F508del mutation and one minimal function mutation.

In the early stages of the FCX-007 program, no children were included. Fibrocell has been testing the potential therapy in adults until enough successful data were collected to justify treating the pediatric population.

ContraVir Pharmaceuticals, Inc. announced that the U.S. FDA has granted orphan drug designation to TXL, an investigational drug candidate for the potential treatment of chronic hepatitis B infection in a pediatric patient population.

The FDA has accepted a supplemental Biologics License Application (sBLA) for Shire’s Cinryze, a therapy that treats recurring swelling attacks in hereditary angioedema (HAE), to expand the already approved indication to include children 6 years and older.

Soleno Therapeutics has completed its End-of-Phase II meeting with the FDA regarding the biopharmaceutical company’s drug, Diazoxide Choline Controlled-Release (DCCR), a therapy used to treat patients with Prader-Willi syndrome (PWS).

GenSight Biologics has published successful results in the Journal of the American Academy of Ophthalmology from their Phase I/II clinical trial and long-term follow-up of GS010, a therapy used to treat patients with Leber Hereditary Optic Neuropathy (LHON).

In a story written by Jackie MacMullan and published on ESPN.com, it was revealed the Hall of Fame basketball player Nate “Tiny” Archibald has been living with amyloidosis.