Rare Diseases
Expert Interview
Advances in testing and therapeutics are improving the lives of patients with Fabry disease
Thanks to robust research efforts, treatment options are expanding and patients are getting their diagnosis earlier – often, when they are...
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.”
Supplement
Spotlight on SMA, Part 2: The Spinal Muscular Atrophy Treatment Landscape
FDA/CDC
FDA OKs new drug for Fabry disease
“The totality of clinical data suggests that Elfabrio has the potential to be a long-lasting therapy.”
Conference Coverage
U.S. incidence, prevalence of myasthenia gravis is rising
These new estimates are “significantly higher” than those from a prior U.S. analysis from 2003.
FDA/CDC
FDA gives fast-track approval to new ALS drug
“The FDA’s approval of Qalsody gives me hope that people living with this rare form of ALS could experience a reduction in decline in strength,...
Latest News
Study compares noninvasive treatments of cutaneous neurofibromas
The epidermis overlying cNF is normal, “so there is no reason to use nonselective or surgical methods and destroy a perfectly good epidermis when...
FDA/CDC
FDA OKs first drug for Rett syndrome
The drug is designed to treat the core symptoms of Rett syndrome by potentially reducing neuroinflammation and supporting synaptic function.
Feature
More data back Guillain-Barré risk with Janssen COVID shot
Observed GBS cases after the Janssen shot were 2 to 3 times greater than expected, based on background rates within 21 and 42 days of vaccination...