February 19, 2021August 24, 2022 Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by ... Read more
February 18, 2021February 18, 2021 High Ferritin Levels Linked to Increased HLH Risk; Testing Urged People with high levels of the iron-storing protein ferritin may be at an increased risk for hemophagocytic lymphohistiocytosis (HLH), a new study suggests. Thus, they ... Read more
February 11, 2021February 11, 2021 Pandemic Won’t Stop Rare Disease Day on Feb. 28 Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness ... Read more
February 4, 2021February 4, 2021 NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many ... Read more
January 28, 2021January 28, 2021 COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the ... Read more
News, syndicated NORD’s Caregiver Respite Program Continues Through Pandemic January 21, 2021January 21, 2021 Caring for a loved one with a rare disease, especially during these uncertain times, demands ...
uncategorized Liver Enzyme Ratio Tied to Higher Mortality Risk in Secondary HLH January 14, 2021January 14, 2021 The proportions of two liver-associated enzymes found in adults with secondary hemophagocytic lymphohistiocytosis (sHLH) is ...
News, syndicated NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum January 11, 2021January 11, 2021 The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences ...
uncategorized Consider Chronic Granulomatous Disease Diagnosis in Children With HLH January 7, 2021January 7, 2021 Physicians should consider chronic granulomatous disease (CGD) in infants and children who present with hemophagocytic lymphohistiocytosis ...
uncategorized Fibrinogen May Be Survival Marker in Secondary HLH December 17, 2020December 17, 2020 Low levels of the blood clotting factor fibrinogen in adults with secondary hemophagocytic lymphohistiocytosis (sHLH) ...
uncategorized ‘Simple’ QuantiFERON‐TB Gold Blood Test May Help in Diagnosing HLH December 10, 2020December 10, 2020 QuantiFERON-TB Gold by Quiagen, a widely used blood test, was found to be helpful in ...
European CHMP Adopts Second Negative Opinion on Gamifant Authorization December 3, 2020December 3, 2020 The European Committee for Medicinal Products for Human Use (CHMP) has recommended the denial of ...
NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting December 3, 2020December 3, 2020 To empower and equip members of the rare disease community to engage state leaders in ...
Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe November 26, 2020November 26, 2020
Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry November 19, 2020November 19, 2020
Tadekinig Alfa Moves Closer to US Market Launch With Manufacturing Deal November 12, 2020November 12, 2020
NORD Hosting ‘RareLaunch’ Workshops to Help Start Rare Disease Non-profits November 5, 2020November 5, 2020
UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner October 29, 2020October 29, 2020 A photo of a bespectacled young boy, his red baseball cap slightly askew as he ...
Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say October 22, 2020October 22, 2020
HSCT of Greater Benefit to Children with Confirmed Familial HLH, Trial Finds October 15, 2020October 15, 2020
Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos October 8, 2020October 8, 2020
Mortality Risk in Pediatric HLH Linked in Study to Impaired Organ Function, Other Factors October 1, 2020October 1, 2020 Hemophagocytic lymphohistiocytosis (HLH) pediatric patients at risk of short-term mortality share certain features, such as ...
Only 10.4% of Suspected HLH Cases Positive for Disease-causing Mutations, Study Finds September 24, 2020September 24, 2020
Ruxolitinib Successfully Treats HLH in Boy With Viral Infection, Case Study Reports September 10, 2020September 10, 2020
News, syndicated Same But Different Photo Contest Celebrates People With Rare Diseases September 3, 2020September 3, 2020 Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is ...
News, syndicated Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input August 27, 2020August 27, 2020 Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed ...
News, syndicated EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US August 20, 2020August 20, 2020 The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to ...
News, syndicated New Streaming Channel Showcases Rare Disease Films August 13, 2020August 13, 2020 When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated ...
uncategorized European CHMP Delivers Negative Opinion of Gamifant; Sobi Requests Re-examination August 6, 2020August 6, 2020 The Committee for Medicinal Products for Human Use (CHMP) recommended denying Sobi‘s marketing authorization request ...
News B-cell Lymphomas May Underlie HLH Development in Rare Cases, Report Shows July 30, 2020July 30, 2020 Although rare, B-cell lymphomas may be the underlying cause of hemophagocytic lymphohistiocytosis (HLH), and complete remission may be achieved ...
uncategorized High-dose Methylprednisolone Successfully Treats HLH, Case Report Shows July 24, 2020July 24, 2020 A case of secondary hemophagocytic lymphohistiocytosis (HLH) — caused by pembrolizumab treatment for lung cancer ...
uncategorized Nivolumab Effective Against Epstein-Barr Virus-Associated HLH, Analysis Suggests July 16, 2020July 16, 2020 The cancer medication nivolumab (marketed as Opdivo) can successfully treat patients with secondary hemophagocytic lymphohistiocytosis (HLH) triggered ...
News, syndicated National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US July 9, 2020July 9, 2020 The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease ...
