Rare Disease Advisor
@raredisease_adv
Trusted knowledge base of practical information and resources focused on treating and diagnosing #RareDisease.
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https://www.rarediseaseadvisor.com/ 17-03-2021 20:37:38
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💡Researchers found that a 12-week home-based exercise program developed for patients with primary biliary cholangitis was safe, well-tolerated, and resulted in improvements in daytime somnolence, fatigue & physical function. 📚 JHEP Reports rarediseaseadvisor.com/news/home-exer…
A new analysis of phase 3 GRIPHON study data evaluated changes in risk status for pulmonary arterial hypertension (PAH) patients using the REVEAL 2.0 risk calculator from the Registry to Evaluate Early and Long-Term PAH Disease Management. Read more: rarediseaseadvisor.com/news/reveal-li…
The Gly103Arg variant of hereditary transthyretin amyloidosis, specific to the Chinese population, presented predominantly with ophthalmological and peripheral nervous system symptoms linked to transthyretin amyloid polyneuropathy. 📚 Frontiers - Neurology bit.ly/4d4nSes
The FDA has accepted KalVista Pharmaceuticals, Inc.’ New Drug Application for #sebetralstat, an oral plasma kallikrein inhibitor designed for the on-demand treatment of hereditary angioedema attacks in individuals over 12 years. bit.ly/3MOIk8h
My story in Rare Disease Advisor on #MIT math whiz Ben Lou, who maintains a perfect 5.0 GPA despite his #spinalmuscularatrophy, and his caregiver mom Jenny Huang. @mdaorg NORD Spinal Muscular Atrophy UK huihua (Jenny) huang #SMA #RareDisease Cure SMA rarediseaseadvisor.com/features/with-…
#Scoliosis surgery seems to have a positive impact on respiratory function and overall satisfaction with posture and physical appearance in patients with spinal muscular atrophy. 📚 Muscle & Nerve Read more: rarediseaseadvisor.com/news/multidisc… #MedNews