Rare Diseases
Rare Diseases Report 2023
The Orphan Drug Act and NORD at their 40th anniversary: Dramatic achievements and ongoing innovation
The movement whose face is ODA and NORD continues to build its legacy. Next? Progress in rare disease care will require an all-in approach to...
Rare Diseases Report 2023
A new chapter for research on treating Huntington’s disease
BY JENNIE SMITH
The past decade has been a contradictory one for research on Huntington’s disease, marked by breakthroughs in the biology...
Rare Diseases Report 2023
Advances in testing and therapeutics are improving the lives of patients with Fabry disease
Treatment options are expanding and patients are getting their diagnosis earlier – often, when they are presymptomatic and treatment has greater...
Rare Diseases Report 2023
Guillain-Barré syndrome: Honing treatment strategies
Classic subtypes of Guillain-Barré syndrome are varying manifestations of a shared disease process, novel insights into the disease indicate. This...
Rare Diseases Report 2023
The dawning age of therapy for Friedreich ataxia
The first therapy to target the underlying pathology of Friedreich ataxia was approved in 2023. Other drug and genetic therapies are in the...
Sponsored Supplement
Spotlight on SMA: The urgent need for early diagnosis in spinal muscular atrophy
Rare Diseases Report 2023
Has prompt diagnosis of amyotrophic lateral sclerosis become urgent?
Optimism is high about improving the survival and care of ALS patients. Neurologists who don’t specialize in ALS can add to the positivity by...
Rare Diseases Report 2023
Emerging therapies in Duchenne and facioscapulohumeral muscular dystrophy
Newly approved and investigational therapies, and enhanced diagnostics, are sparking optimism about treating MD especially Duchenne and...
Latest News
West Nile infections rising in the U.S.
West Nile Virus is the leading cause of mosquito-borne disease in the continental United States.
Conference Coverage
Gene therapy promising for reversal of hereditary vision loss
The results confirm in a “real-life setting” the efficacy and safety of the treatment.
FDA/CDC
Myasthenia gravis drug gets FDA nod
Rozanolixizumab is the first FDA-approved treatment for adults with both subtypes.
Literature Review
Can a repurposed Parkinson’s drug slow ALS progression?
One expert interviewed for this article said the study “falls short of being a credible 1/2a clinical trial.”