Krista Rossi

Morie Gertz, MD, discusses a new treatment for hATTR amyloidosis patients, inotersen, as it makes its way towards FDA approval.

Researchers at Penn Medicine have found patients with more severe forms of neurodegenerative diseases possess more co-pathologies.

Updated phase 1 data assessing indoximod plus front-line radiation and maintenance chemotherapy for the treatment of pediatric patients with DIPG proves promising.

Researchers from UT Southwestern Medical Center have identified 29 genes thought to contribute to rhabdomyosarcoma using the iExCN algorithm, a novel hybrid technology.

A new drug under development for the treatment of paroxysmal nocturnal hemoglobinuria has been shown to be beneficial in participants enrolled 2 ongoing phase 1b trials.

A recent phase 1 study evaluating ARO-AAT for the treatment of a rare genetic liver disease associated with alpha-1 antitrypsin (AAT) deficiency boasts positive initial clinical data.

New study finds that half of all young children with PFIC will need a liver transplant before age 10.

Teleconsulting technology through the European Network for Rare Adult Solid Cancer (EURACAN) will better help rare adult solid cancer patients achieve appropriate diagnoses and treatments.

New Addison's Disease Information System (ADIS) device assists Addison disease patients and providers by providing access to web-based clinical management information.

The FDA has granted Fast Track Designation for Cyprium Therapeutics’ CUTX-101, developed for the treatment of certain patients with classic Menkes disease.

The FDA has cleared commencement for Retrotope’s open-label phase 2/3 clinical trial of its investigational drug RT001 to evaluate its efficacy and safety in patients with infantile neuroaxonal dystrophy (INAD).

Positive developmental updates on the investigational BTK inhibitor, zanubrutinib, as a treatment for Waldenström macroglobulinemia were recently released.

The FDA has granted orphan drug designation to Oncology Venture AB for their oral phase 3 multi tyrosine kinase inhibitor (previously TKI258), dovitinib DRP, for the treatment of adenoid cystic carcinoma (AdCC).

Multiple hematology organizations have come together to develop state-of-the-art guidelines for the diagnosis and management of von Willebrand Disease.

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

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.

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).

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.