| 3/31/2021 |
Uplifting Athletes Presents $140,000 in Rare Disease Research Grants at 2021 Young Investigator Draft |
mysanantonio.com |
Uplifting Athletes Presents $140,000 in Rare Disease Research Grants at 2021 Young Investigator Draft PRWebSports and Science Team Up to Honor Emerging Medical Researchers at Annual Celebration Modeled After NFL Draft PHILADELPHIAUplifting Athletes , a nonprofit organization serving the Rare Disease Community, awarded $140,000 in medical research grants last night at its Young Investigator Draft Presented by CSL Behring . Seven promising young researchers received unrestricted $20,000 grants to support ... |
| 3/25/2021 |
Second Phase 3 Study of Pamrevlumab Recruiting Boys With DMD |
bionewsfeeds.com |
... muscular dystrophy (DMD) who are able to walk. The trial, LELANTOS-2 (NCT04632940), is enrolling approximately 70 participants ages 6 to 12 at three U.S. sites — Arkansas Children’s Hospital in Little Rock, Rare Disease Research Center in Atlanta, Georgia, and University of Massachusetts Memorial Medical Center in Worcester. “The initiation of LELANTOS-2, the second Phase 3 clinical†... |
| 3/1/2021 |
Focus on Patients to Boost Rare Disease Research Success |
worldwide.com |
Focus on Patients to Boost Rare Disease Research Success Focus on Patients to Boost Rare Disease Research Success By The Editors of Uncommon Conversations, March 1, 2021Far too often, CROs plan a rare disease trial and then go looking for patients to recruit. At Worldwide, we believe the best approach to rare disease research is the other way around: First, find your patients. Talk to them. Understand their experience ... |
| 2/26/2021 |
Rare Disease Day and Licensing Opportunities |
National Institutes of Health |
... and the public. It is observed in 103 countries with thousands of events occurring. On March 1st, NCATS and the NIH Clinical Center will host their Rare Disease Day conference to highlight NIH-supported rare disease research and development, share the latest rare disease information, and share patients’ stories. The event will feature a myriad of speakers such as the NIH Director, patients and their families, and rare disease ... |
| 2/26/2021 |
PPD Lights Up Headquarters Downtown for 2021 Rare Disease Day |
financialbuzz.com |
... patients and families living with rare diseases every day. It also allows PPD to help educate the general public, both locally and globally, on rare diseases. For more information on PPD’s commitment to rare disease research , please visit https://www.ppd.com/therapeutic-expertise/rare-diseases .WHEN:Friday, Feb. 26, 2021. Illumination begins shortly after dusk and will end at 11:30 p.m.(Please note: Rare Disease Day officially is observed on ... |
| 2/26/2021 |
Factor IX Assay Discrepancies in the Setting of Liver Gene Therapy Using a Hyperfunctional Variant Factor IX‚ÄêPadua |
wiley.com |
Accepted Articles ORIGINAL ARTICLE Factor IX Assay Discrepancies in the Setting of Liver Gene Therapy Using a Hyperfunctional Variant Factor IX‐Padua Corresponding AuthorPfizer Inc, Cambridge, MA, USACorresponding Author: John E. Murphy, PhD, Vice President, Biology, Rare Disease Research , Pfizer Inc., 610 Main Street, Cambridge MA 02139., Tel: 617‐945‐4872E‐mail:Children’s Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA ... |
| 2/2/2021 |
Twin Anemia Polycythemia Sequence Day – Recognizing TAPS Research Worldwide |
issuewire.com |
Celebrating 15 years of Rare Disease Research taps twins 2Almere, Flevoland Feb 2, March 3, 2021, marks 15 years since the naming of Twin Anemia Polycythemia Sequence, a rare disease affecting identical twins. On this date, the TAPS Support Foundation will be recognizing the work of researchers worldwide, who have spent their time dedicated to discovering more about TAPS and raising awareness throughout February on vital aspects of this ... |
| 1/28/2021 |
Patient Data Registry Approach May Enhance Rare Disease Research |
HealthIT Analytics |
Tools & Strategies News Patient Data Registry Approach May Enhance Rare Disease Research A new type of patient data registry could provide researchers with quality information on rare diseases, accelerating treatment development. Source: ThinkstockByJanuary 28, 2021 - Researchers at the Perelman School of Medicine at the University of Pennsylvania have designed a new kind of patient data registry to advance rare disease research.In a paper published in Cell Reports ... |
| 1/8/2021 |
Sarepta gene therapy misses goal in key muscular dystrophy study |
biopharmadive.com |
... doctors who hope the therapy could be a one-time treatment for the fatal genetic condition.The result is from one of the most closely watched clinical trials in gene therapy and, more broadly, rare disease research . Sarepta has been racing to prove its gene therapy can help halt and even reverse the steady muscle degeneration brought on by the disease. The Cambridge, Massachusetts-based biotech company's setback came the ... |
| 11/23/2020 |
FOXG1 Research Foundation to Pioneer a Machine Learning Approach to Accelerate Rare Disease Research with Support From the Chan Zuckerberg Initiative | Business & Finance | heraldchronicle.com |
PR Newswire |
... and retention. Costs to patient organizations for studies can exceed $10,000 per subject per year and participants typically do not have access to study results — all limiting patients' access to and engagement in rare disease research.Thanks to two years of planning and this grant from CZI, the FOXG1 Research Foundation is launching a groundbreaking study using technology and machine learning to supplement the current NHS model by digitally ... |
| 11/21/2020 |
First-Ever Treatment for Rare Rapid-Aging Disease Progeria Receives U.S. FDA Approval |
BioSpace |
... Drug Administration (FDA) approval of Zokinvy‚Ñ¢ (lonafarnib), for the treatment of Progeria and processing-deficient Progeroid Laminopathies (PL). Progeria is an ultra-rare, fatal pediatric rapid-aging disease. PRF, a pioneer in the rare disease research foundation space, has led Zokinvy clinical trial research since 2007.Zokinvy is a farnesyltransferase inhibitor (FTI) that has shown survival benefit in children with Progeria. Data based on information from the PRF International ... |
| 11/20/2020 |
First-Ever Treatment for Rare Rapid-Aging Disease Progeria Receives U.S. FDA Approval |
ask.com |
... Drug Administration (FDA) approval of Zokinvy‚Ñ¢ (lonafarnib), for the treatment of Progeria and processing-deficient Progeroid Laminopathies (PL). Progeria is an ultra-rare, fatal pediatric rapid-aging disease. PRF, a pioneer in the rare disease research foundation space, has led Zokinvy clinical trial research since 2007.Zokinvy is a farnesyltransferase inhibitor (FTI) that has shown survival benefit in children with Progeria. Data based on information from the PRF International ... |
| 11/13/2020 |
New Treatments Offer Hope for Spinal Muscular Atrophy Patients |
usmedicine.com |
... and Drug Administration and at least five more have begun clinical trials.The dramatic shift may be attributed to two transformative trends. Turbocharged by social media platforms, efforts by patients, caregivers, advocacy organizations and rare disease research foundations to connect with each other, give greater voice to their issues, and attract researchers and investment have seen unprecedented success over the last decade. At the same time, breakthroughs in genetic analysis ... |
| 11/10/2020 |
Pfizer invests in Homology and adds to its gene therapy ambitions |
biopharmadive.com |
... is the latest by Pfizer to add to its growing gene therapy presence, which has been built through a steady stream of acquisitions and licensing deals. Seng Cheng, the chief scientific officer of Pfizer's rare disease research unit, will join Homology's scientific advisory board to help with development of HMI-102 for adults with PKU and HMI-103 for pediatric patients. Dive Insight:The investment gives Pfizer right of first ... |
| 10/26/2020 |
CANbridge Pharmaceuticals Appoints Head of Global Research |
intellasia.net |
... be based at the CANbridge US headquarters in Cambridge, Massachusetts.“Yunxiang comes to CANbridge on the heels of our second rare disease gene therapy Research Agreement with UMass Medical School, underscoring our commitment to rare disease research and developing a world-class CANbridge research capability, particularly in biologics and gene therapy,” said James Xue, PhD, CANbridge Founder, Chairman and CEO. “He will be instrumental in expanding our US-based global ... |
| 10/26/2020 |
CANbridge Pharmaceuticals Appoints Head of Global Research |
BioSpace |
Yunxiang comes to CANbridge on the heels of our second rare disease gene therapy Research Agreement with UMass Medical School, underscoring our commitment to rare disease research and developing a world-class CANbridge research capability, particularly in biologics and gene therapy,” said James Xue, PhD, CANbridge Founder, Chairman and CEO. “He will be instrumental in expanding our US-based global research programs and driving our strategy of in-China development ... |
| 9/29/2020 |
Treatment-resistant rare diseases: exploring landmark drug approvals in 2020 |
nridigital.com |
... who so desperately need them.” Barriers to drug developmentThere are multiple challenges involved with rare disease drug development. With experience working with the US National Organization for Rare Disorders (NORD) and directly in rare disease research , Garrison sees the main challenges being at pre-clinical stages.In pre-clinical stages, Garrison notes that the difficulty of modelling rare diseases is a major barrier.“An appropriate model that represents the ... |
| 9/10/2020 |
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malvern-online.com |
... research forward and we’re proud to present these data at the ASBMR 2020 Annual Meeting,” said Howard Mayer, M.D., Executive Vice President and Head of Research and Development at Ipsen. “Collaboration is critical with rare disease research and development and we look forward to continuing to work with key thought leaders, clinicians, the patient advocacy community, and regulatory authorities as we build on our research and develop potential therapeutic agents ... |
| 9/10/2020 |
2020 NORD Summit to Feature Leading Experts in Public Policy, Patient Advocacy, Rare Disease Research and Regulatory Science |
PR Newswire |
... disease community and connecting to discuss the most current and critical topics related to rare diseases and orphan products. This year's theme, "Entering a New Era," refers to the current climate of innovation in rare disease research, evolving regulatory science and the need to address global public health challenges. Throughout the Summit, the focus will be on maintaining and advancing innovation while addressing current challenges. Top sponsors of the 2020 ... |
| 9/10/2020 |
2020 NORD Summit to Feature Leading Experts in Public Policy, Patient Advocacy, Rare Disease Research and Regulatory Science |
BioSpace |
WASHINGTON , Sept. 10, 2020 /PRNewswire/ -- At a time when it is vitally important for the rare disease community to come together, the National Organization for Rare Disorders (NORD®) has announced a program of broad-ranging topics with speakers of unparalleled expertise for the 2020 NORD Rare Diseases and Orphan Products Breakthrough Summit , taking place virtually October 8-9 ... |