RDR Alert®

Orphan drug designation has been granted to CPI-613 for the treatment of Burkitt lymphoma as a phase 2 trial commences for the drug.

Individuals with vitamin D deficiency may be at greater risk of interstitial lung disease, a rare disease that causes scarring of the lungs.

The US Food and Drug Administration (FDA) has approved encorafenib and binimetinib (BRAFTOVI and MEKTOVI) in combination for patients with unresectable or metastatic melanoma with a BRAF V600E or V600K mutation as detected by an FDA-approved test.

Positive top-line data from Part A of a recent phase 3 trial evaluating voxelotor for the treatment of sickle cell disease have been released.

Updated data on MoveDMD, a phase 2, open-label extension trial of edasalonexent in boys diagnosed with Duchenne muscular dystrophy (DMD), reports positive results.

Takami Sato, MD, PhD, Department of Medical Oncology, Kimmel Cancer Center, Thomas Jefferson University, discusses new treatment for metastatic uveal melanoma: IMCgp100.

Rigel Pharmaceuticals has recently selected US Bioservices to dispense fostamatinib disodium hexahydrate tablets, for the treatment of adult patients with chronic immune thrombocytopenia (ITP).

The 2018 American Society of Clinical Oncology (ASCO) Annual Meeting held in Chicago, Illinois, June 1-5, 2018, featured data from numerous studies and advancements being made in rare cancers. Here are 5 key takeaways from the meeting.

New findings suggest that riluzole prolongs survival in the final clinical stage of amyotrophic lateral sclerosis (ALS).

In a study conducted at Children's Hospital Los Angeles (CHLA), less-toxic treatment regimens for mixed phenotype acute leukemia (MPAL) have been found to coincide with disease remission.

The FDA has approved cannabidiol (Epidiolex) [CBD] oral solution in patients aged 2 years and older for the treatment of severe and rare types of epilepsy, Lennox-Gastaut syndrome and Dravet syndrome.

The FDA has granted Priority Review for a supplemental New Drug Application (sNDA) for the company’s Waldenström's macroglobulinemia combination therapy, ibrutinib (IMBRUVICA) in combination with rituximab (RITUXAN).

Stay up-to-date on the latest rare disease news by reading the top 5 articles of the week.

The first patient has been enrolled in a pivotal phase 3 clinical trial evaluating the safety and efficacy of an epithelium-on (epi-on) corneal collagen cross-linking procedure to treat progressive keratoconus.

The FDA has granted Orphan Drug Designation to its product, PCS499, for treatment of necrobiosis lipoidica.

Positive preliminary results from the Sarepta Therapeutics, Inc phase 1/2a gene therapy clinical trial assessing AAVrh74.MHCK7.micro-Dystrophin vector in individuals with Duchenne muscular dystrophy (DMD) indicate outstanding promise.

hATTR compass program will empower people with accurate genetic information so they can make informed decisions about their healthcare.

The FDA has approved a label expansion for C1 esterase inhibitor [human] (CINRYZE) as a preventative measure for pediatric angioedema attacks in children aged 6 years and older with hereditary angioedema (HAE).

Nicholas J Robert, MD, discusses treatment challenges associated with rare cancers like Merkel cell carcinoma.

Updated 24-week data was released from patients treated with the 900 mg dose of voxelotor in the ongoing HOPE-KIDS 1 Study, a phase 2a open-label study in adolescents ages 6 to 17 years with sickle cell disease (SCD).

Updated interim data from Affimed NV’s phase 1b dose-escalation study evaluating AFM13 for the treatment of relapsed/refractory Hodgkin Lymphoma were released at the 23rd Congress of the European Hematology Association.

Alexion Pharmaceuticals, Inc. has submitted a BLA to the FDA for approval of ALXN1210 for the treatment of patients with paroxysmal nocturnal hemoglobinuria.

Sickle Cell Disease Association of America (SCDAA) and Emmaus Life Sciences partner in an effort to combat sickle cell disease.

Make-A-Wish New Jersey celebrated 35 years of wish granting in the Garden State at its annual Gala Thursday night, filled with celebrity surprises, a wish granted, and a historic first-time meeting, and musical entertainment by Max Weinberg of the E-Street Band and Friends.

In honor of World Sickle Cell Awareness Day, we’ve rounded up the big treatment advances made this year thus far for sickle cell disease.