Alfred Santiago the third needs your help. The 2-year-old was born with a rare congenital disorder where a part of his brain is shrinking over time. His father, Alfred Santiago Jr., who works and lives part-time in the Bronx, has found a California doctor who will perform a stem-cell surgery...
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BROOKLYN, Mich. – Full of life and full of love. That’s who four year-old Jaxon Meschke is. “All my friends! I love mom. I love dad and baby brother,” said four-year old Jaxon. But it’s his perseverance that stands out to his parents Brian and Chelsea Meschke most. “He really...
EATONS HILL — An Eatons Hill family knows the daily battles of managing life with cystic fibrosis. Alison and Ben Perkin are the proud parents of four children, two of whom have cystic fibrosis (CF), the most common incurable genetic condition that substantially shortens life expectancy. Mrs Perkin said her...
SAN CARLOS, Calif. — BeOne Medicines Ltd. (Nasdaq: ONC; HKEX: 06160; SSE: 688235), a global oncology company, reaffirms its position as the leader in chronic lymphocytic leukemia (CLL) innovation by showcasing the depth, quality, and momentum of its hematology portfolio at the 67th ASH Annual Meeting and Exposition in Orlando, Florida. The...
CAMBRIDGE, Mass. & BEIJING – BeiGene (NASDAQ: BGNE; HKEX: 06160) announced positive results from a planned interim analysis of the Phase 3 ALPINE trial comparing BRUKINSA® (zanubrutinib) against ibrutinib in adults with relapsed or refractory (R/R) chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). BRUKINSA met the primary endpoint of the trial, demonstrating...
In 2008, Sandra Bedrosian Sermone gave birth to twin boys, Tony and Rocco. Early on, Tony was in and out of the hospital for four years with no diagnosis as he lost the ability to swallow, walk and talk. At 4, he was diagnosed with a number of conditions including...
Boston, Mass. — Childhood interstitial lung disease (chILD) is a group of rare, genetic lung disorders affecting infants and children who typically present with a range of symptoms, such as shortness of breath, rapid breathing and coughing. While many causal genes have been identified for chILD, understanding its pathogenesis has...
Novato, Calif. – Restricting calories is known to improve health and increase lifespan, but much of how it does so remains a mystery, especially in regard to how it protects the brain. Buck scientists have uncovered a role for a gene called OXR1 that is necessary for the lifespan extension...
Digital communities have become support networks for patients of rare diseases, as well as professionals. They boost access to research, encourage advocacy, and aid evidence-gathering When Lucinda Andrews learned that her newborn son was only the 16th person in the world known to be diagnosed with an ultra-rare genetic disorder, she...
STOCKHOLM, Sweden – Vicore Pharma Holding AB (STO:VICO), unlocking the potential of a novel class of drugs, angiotensin II type 2 receptor agonists (ATRAGs), today announced that Japan’s Ministry of Health, Labor and Welfare (MHLW) has granted Orphan Drug designation to buloxibutid for the treatment of idiopathic pulmonary fibrosis (IPF)....