News
Conferences
Videos
Case Studies
Insights
Interviews
Conditions
Partners
Resource Guide
Newsletter Signup
For Patients
Latest News
View More >>
Biallelic Mutations Causing Rare Pediatric Brain Disorder VARS
Insights on Rare MICPCH Syndrome Could Help with Autism, Epilepsy
FDA Grants Priority Review to 2 Cancer Investigational Drugs
Age, Sex Are Risk Factors for Arrhythmic Events in Long QT Syndrome
Experimental ALS Therapy Uses Antibodies to Target TDP-43 protein
New Therapeutic Target for Myeloproliferative Neoplasm
Huntington's Disease
Oncology
Neurology
MPNs
Top 5 Rare Disease News of the Week—September 9, 2018
Top 5 Rare Disease News of the Week—July 8, 2018
Genomic Link Discovered Between Rheumatoid Arthritis and Huntington
PTC's Splicing Technology Makes Its Way to Huntington's Disease
Why is Cancer Uncommon in Huntington's Disease Patients?
New Data Suggest Enzyme Blockage Could Fight HD
Five Facts About: Huntington's Disease [Infographic]
New Pathway Identified in Huntington's Disease Cells
View More
>>
Targeting an Immune Regulating Protein May Help Halt Glioblastoma Progression
Engineered Cancer Antivirus Has Antisarcoma Effect
Therapeutic Target for Medulloblastoma Identified in Mice Models
FDA Approves Ibrutinib/Obinutuzumab for Treatment-Naive Patients with Chronic Lymphocytic Leukemia
Refining Leukemia Classification for More Accurate Diagnoses
FDA Approves Trastuzumab for Metastatic Gastric/Gastroesophageal Junction Adenocarcinoma
FDA Approves Hepatocellular Carcinoma Therapy, Cabozantinib
Senescent Cell Research Shows Potential for Idiopathic Pulmonary Fibrosis
View More
>>
Insights on Rare MICPCH Syndrome Could Help with Autism, Epilepsy
Acute Flaccid Myelitis Paralysis Reversed in New Surgery
Muscular Dystrophy Treatment with CRISPR Technology on the Way
Dipanjan Basu, PhD, Highlights Potential of Omipalisib for Neurocutaneous Melanocytosis
Lambert-Eaton Myasthenic Syndrome (LEMS) Gains First FDA-Approved Treatment
New PKU Drug Already Benefiting Adults
Clobazam (SYMPAZAN) Oral Film Approved by FDA for Lennox-Gastaut Syndrome
FDA Grants Fast Track Designation to A4250 for Progressive Familial Intrahepatic Cholestasis
View More
>>
New Therapeutic Target for Myeloproliferative Neoplasm
Hydroxyurea Therapy Found Effective for Myeloproliferative Neoplasms
Males with MPN Have Inferior Survival Compared to Females
Daily Smokers Have a More than 3 Times Increased Risk for MPN
Young Myeloproliferative Neoplasms Patients Have Longer Survival than Older Counterparts
Using Interferon‐α2 & Ruxolitinib Combination Therapy to Treat Myeloproliferative Neoplasms
Myeloproliferative Neoplasms Impair Patient Employment Status & Work Productivity
Gene Found to Enable Deadly Myelodysplastic Syndromes
View More
>>
Latest Videos
Interviews
Jasmine Zain, MD, Highlights Current & Upcoming T-Cell Lymphoma Treatments
Michael Jordan, MD, Differentiates Emapalumab Treatment in Pediatric Patients Versus Adults with HLH
Germ Cell Tumors Have the Potential to Become Leukemia
AI: The Future of Rare Disease Treatment?
View More >>
Insights
Genetic Counselors and Fabry Disease
Fabry Disease - What a Clinician Needs to Know
Fabry Disease: An X-Linked Genetic Disease
Newborn Screening and Fabry Disease: What We Know and Don't Know
View More >>
Case Studies
Polycythemia Vera Case Study - Final Thoughts on Ruxolitinib
The Safety and Efficacy of Ruxolitinib in Polycythemia Vera
10 Questions to Ask Polycythemia Vera Patients
Treating Polycythemia Vera Patients Refractory to Hydroxyurea
View More >>
Conference Coverage
Mohamad Mohty, MD, PhD, Discusses Cytotoxic T Lymphocytes as Potential EBVPTLD Therapy
Peter Turecek, PhD, Explains How Medical Devices May Benefit Patients with Hemophilia
Ruxolitinib Therapy Benefits Patients with Chronic Neutrophilic Leukemia
Hydroxyurea Therapy Found Effective for Myeloproliferative Neoplasms
Pemigatinib Shows Promising Efficacy as Myeloid/Lymphoid Neoplasms Treatment
Nizar Bahlis, MD, Recaps Follow-Up Data on Combo Therapy for R/R Multiple Myeloma
