Rare disease ruined his life
A TEEN has spent the last 10 years in a wheelchair after he was diagnosed with a rare genetic muscular disease known as ...
STAT3d
In a bid to treat their children’s rare diseases, these three parents founded companies
At #STATSummit, three parents discussed the challenges they've faced trying to find treatments for their children with rare ...
The Peninsula3d
Sidra Medicine pioneers gene therapy in Qatar for children with rare genetic disorders
Sidra Medicine s Genetic and Genomic Medicine clinic is providing life altering gene therapy for rare genetic disorders. In a ...
stockhead4d
Dr Boreham’s Crucible: Does this biotech have the horsepower to become ‘the next Neuren’?
While success is far from guaranteed in bio-land, with this week’s raising, Percheron has the horsepower to have a decent ...
Researchers identify possible treatment for rare disorder CTNNB1 syndrome
Researchers at Tufts University School of Medicine and the Graduate School of Biomedical Sciences (GSBS) have identified a ...
Nido Biosciences Announces Orphan Drug Designation for Lead Clinical Candidate to Treat Spinal and Bulbar Muscular Atrophy
Nido Biosciences (Nido Bio), a clinical stage company developing precision medicines for debilitating neurological diseases, ...
$3.2 million per dose Elevidys fails to meet primary endpoint in phase 3 trial
A Phase 3 clinical trial investigating Elevidys (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular ...
Refusing to let rare disease define her, Rashmirekha Bhuyan in Assam leads extraordinary life
ASSAM: Rashmirekha Bhuyan knew at an early age that eventually the spinal muscular atrophy (SMA) would confine her to the ...
Convict-era disease found in Australia
A case of the rare disease has been detected in Australia, in what some doctors are calling the re-emergence of a ...
Medscape6d
Myasthenia Gravis: Similar Symptoms in Relatives Raise Question of Genes
In one of the case reports, a 75-year-old White man with hereditary coagulopathy presented with myasthenic crisis in the setting of acute pulmonary embolism. Chronic symptoms included diplopia, ptosis ...
The Globe and Mail3d
Spinal Muscular Atrophy Pipeline Therapeutics, Assessment, Companies, Products, Unmet Needs, Market Drivers and Barriers
Spinal Muscular Atrophy Pipeline Outlook Apitegromab is a selective ... The US Food and Drug Administration (FDA) has granted ...