OND

Limb-Girdle Muscular Dystrophy Leaders Advance Collaborative Dialogue to Accelerate Drug Development to Address the Urgent Unmet Need in Limb-Girdle Muscular Dystrophy

Retrieved on: 
Tuesday, February 27, 2024
LGMD, Nationwide Children's Hospital, Analysis Group, OND, FDA, Clinical trial, Food, Drug development, Sarepta Therapeutics, Virginia Commonwealth University, Hospital, Treatment, Senior advisor, Documentation, University, CDER, Therapy, Keynote, Patient, Science, Limb–girdle muscular dystrophy

The meeting focused on six LGMD subtypes: 2A/R1, 2B/R2, 2C/R5, 2D/R3, 2E/R4, and 2I/R9.

Key Points: 
  • The meeting focused on six LGMD subtypes: 2A/R1, 2B/R2, 2C/R5, 2D/R3, 2E/R4, and 2I/R9.
  • We are thrilled that the workshop advanced a collaborative dialogue and brought people together from every facet of the drug development process to drive progress for patients.
  • This was truly a monumental event for the LGMD community and a model for others to follow in the future."
  • In-depth documentation of the workshop's findings and next steps is in progress and will be shared when available.

Limb-Girdle Muscular Dystrophy Scientific Leaders - Together With the FDA - Will Come Together for a Drug Development Workshop on February 8, 2024

Retrieved on: 
Wednesday, December 13, 2023
Food, OND, Therapy, Hospital, CBER, Patient, Nationwide Children's Hospital, Virginia Commonwealth University, FDA, CDER, Caregiver, Limb–girdle muscular dystrophy, University, Muscular dystrophy, Drug development, Pharmaceutical industry, Medical device, LGMD, Research

ROCKVILLE, Md., Dec. 13, 2023 /PRNewswire/ -- On February 8, 2024, The Speak Foundation, a patient-led 501(c)(3) organization for Limb-Girdle Muscular Dystrophy (LGMD), will be convening a multi-stakeholder LGMD Scientific Drug Development Workshop in Rockville, Maryland.

Key Points: 
  • ROCKVILLE, Md., Dec. 13, 2023 /PRNewswire/ -- On February 8, 2024, The Speak Foundation, a patient-led 501(c)(3) organization for Limb-Girdle Muscular Dystrophy (LGMD), will be convening a multi-stakeholder LGMD Scientific Drug Development Workshop in Rockville, Maryland.
  • The workshop will continue to drive the momentum for the sense of urgency needed by the patient community to accelerate LGMD drug development.
  • "This workshop is a continuing step to work with the FDA and LGMD scientific leaders on pushing critical drug development forward.
  • The LGMD Scientific Workshop will be a focused discussion with clinicians, scientific researchers, drug developers, regulators, patients and patient advocates on the characteristics of LGMD that drive drug development programs.

Sarah McGarry, M.D., Senior FDA Official in CDER and CBER, Joins Greenleaf Health

Retrieved on: 
Wednesday, November 8, 2023
FDA, COVID-19, Public Policy, Consulting, Pharmaceutical, Professional Services, Medical Devices, Infectious Diseases, Government, Science, Biotechnology, AIDS, Other Science, Health, Research, Georgetown University School of Medicine, Bioinformatics, CBER, Center for Biologics Evaluation and Research, Policy, Food, Infection, Duke University, SARS-CoV-2, Center for Drug Evaluation and Research, OND, HIV, CDER, Ritonavir, Senior, Marketing, Internal medicine, China Biologic Products, Associate, Food and Drug Administration, Pharmaceutical industry

Greenleaf Health, Inc. (Greenleaf), a leading consulting firm for companies regulated by the Food and Drug Administration (FDA), is pleased to announce that Sarah McGarry, M.D., has joined as Senior Vice President, Drug and Biological Products.

Key Points: 
  • Greenleaf Health, Inc. (Greenleaf), a leading consulting firm for companies regulated by the Food and Drug Administration (FDA), is pleased to announce that Sarah McGarry, M.D., has joined as Senior Vice President, Drug and Biological Products.
  • Throughout her 18-year FDA service, Sarah held multiple leadership, review, and advisory positions within the Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER).
  • Her knowledge of the FDA review process and regulatory policy will enrich the strategic and scientific guidance that Greenleaf provides to clients.
  • from Georgetown University Medical School and completed a residency in internal medicine and a fellowship in infectious diseases at Duke University.

Cytokinetics Reports Third Quarter 2023 Financial Results

Retrieved on: 
Thursday, November 2, 2023
Randomness, FDA, National Medical Products Administration, Standard of care, Research, Heart failure, Manuscript, American College, NMPA, Food, Center for Drug Evaluation and Research, Pivotal, LVEF, OND, Symantec Endpoint Protection, Safety, Cytokinetics, Organization, MAD, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Journal of Cardiac Failure, Obstruction, SAD, Journal, Cardiology, CRL, Disopyramide, ALS, Clinical trial, CDE, EMA, Șaeș, Assessment, NDA, Investment, LVOT, Plasma protein binding, Pharmaceutical industry, Medical device, Patient, Pharmacokinetics, Pharmacology, HCM, EU

SOUTH SAN FRANCISCO, Calif., Nov. 02, 2023 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) reported financial results for the third quarter of 2023. Net loss for the third quarter was $129.4 million, or $1.35 per share, compared to net loss for the third quarter of 2022 of $142.3 million, or $1.52 per share. Cash, cash equivalents and investments totaled $554.7 million on September 30, 2023.

Key Points: 
  • On Track for Topline Results from SEQUOIA-HCM,
    a Pivotal Phase 3 Clinical Trial of Aficamten in Obstructive HCM,
    the Open-Label Extension Study of Aficamten,
    SOUTH SAN FRANCISCO, Calif., Nov. 02, 2023 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) reported financial results for the third quarter of 2023.
  • Net loss for the third quarter was $129.4 million, or $1.35 per share, compared to net loss for the third quarter of 2022 of $142.3 million, or $1.52 per share.
  • “During the third quarter we made considerable progress across our specialty cardiology franchise, with aficamten remaining our top priority.
  • During the quarter we also started ACACIA-HCM, a pivotal Phase 3 clinical trial of aficamten in patients with non-obstructive HCM.

LibDib Partners with Gorilla Shipper to Offer Special Maker Pricing and Discounts

Retrieved on: 
Tuesday, October 17, 2023
Packaging, Retail, Transport, Logistics, Supply Chain Management, Wine & Spirits, Manufacturing, Beer, Partnership, FedEx, Condiment, Gold–silver–bronze command structure, Drink, Wine, Gold, OND, Cargo

As part of the agreement, Gorilla Shipper is now offering LibDib Makers significant levels of pricing discounts.

Key Points: 
  • As part of the agreement, Gorilla Shipper is now offering LibDib Makers significant levels of pricing discounts.
  • Makers can use the discounted packaging not only for LibDib shipments, but across their entire business.
  • When using Gorilla Shipper, Makers can expect high-quality, eco-friendly, reliable products, all made in the U.S.A., including pulp to foam shippers, and the Gorilla Shipper patented Hexabox™ corrugated shippers.
  • “We are thrilled to partner with an innovator like LibDib,” said Maria Schoeller, General Manager of Gorilla Shipper.

Yr heriau'n wynebu Rhun ap Iorwerth cyn yr etholiadau nesaf

Retrieved on: 
Monday, July 17, 2023
Senedd, AR, SNP, Fel, OND, Synthetic rubber, Cardboard, Liquor, European

Blaenoriaeth ddi-oed, sydd wedi ei chydnabod gan ap Iorwerth wrth ddod i'r swydd, yw gweithredu 82 argymhelliad yn yr adroddiad.

Key Points: 
  • Blaenoriaeth ddi-oed, sydd wedi ei chydnabod gan ap Iorwerth wrth ddod i'r swydd, yw gweithredu 82 argymhelliad yn yr adroddiad.
  • Er mwyn sicrhau enillion yn yr etholiadau hynny, mae gofyn am fwy na diwygio trefnidaethol a blaengynllunio etholiadol trylwyr.
  • Yr awgrym yn ôl yr arolygon barn ydy taw cyfyngedig fydd unrhyw ennill tir yn yr etholiad cyffredinol nesaf.

Annibyniaeth i Gymru

    • Drwy wneud hynny, roedd yn mabwysiadu yr un math o strategaeth â llawer o bleidiau a mudiadau o blaid annibyniaeth ar draws Ewrop.
    • Y casgliad oedd bod galwadau am annibyniaeth wedi cynyddu, gyda mwy o bwyslais ar gyflwyno achos cadarnhaol dros greu gwladwriaeth newydd.
    • Eto'i gyd, lleiafrif o bleidleiswyr sy'n cefnogi annibyniaeth gyda diwygio cyfansoddiadol yn isel ar y rhestr o faterion sy'n bwysig i bobl.

Esiampl yr Alban

    • Dangosodd methiant ymdrechion Plaid Genedlaethol yr Alban (yr SNP) i sicrhau'r hawl gyfreithiol i gynnal refferendwm arall ar annibyniaeth nad oes llwybr hawdd i'r rhai sydd am adael y DG.
    • A hynny er gwaetha'r trafferthion sy'n wynebu Llywodraeth Cymru yn y meysydd sydd o dan ei adain fel y gwasanaeth iechyd.
    • Wrth gwrs, y tebygrwydd hefyd yw y bydd Llafur Cymru yn ymladd yn yr etholiad nesaf i'r Senedd gydag arweinydd newydd wrth y llyw.
    • Bydd natur yr ornest yn wahanol hefyd wrth i'r diwygiadau i'r Senedd gael eu rhoi ar waith gan gynyddu'r nifer o aelodau o 60 i 96 a chyflwyno system etholiadol newydd.

Mae’n bryd i ni ailfeddwl beth yw gwyddoniaeth dinasyddion

Retrieved on: 
Saturday, June 10, 2023
AER, Blaen-y-Maes, STS, European, Wales, George, AR, BYD, OND, Aviva Investors, Computer data storage, Cardboard, Liquor, Electric motor

Mae gwyddoniaeth dinasyddion yn ddull poblogaidd o gasglu data ar gyfer gwyddonwyr naturiol a chymdeithasol, ac mae nifer y prosiectau a'r cyhoeddiadau a gynhyrchir yn tyfu o flwyddyn i flwyddyn.

Key Points: 
  • Mae gwyddoniaeth dinasyddion yn ddull poblogaidd o gasglu data ar gyfer gwyddonwyr naturiol a chymdeithasol, ac mae nifer y prosiectau a'r cyhoeddiadau a gynhyrchir yn tyfu o flwyddyn i flwyddyn.
  • Mae prosiect gwyddoniaeth dinasyddion nodweddiadol yn defnyddio gwirfoddolwyr i gasglu data a fyddai, fel arall, yn anfforddiadwy neu'n anhygyrch.

Beth yw gwyddoniaeth?

    • Rydym yn gweithio o fewn astudiaethau gwyddoniaeth a thechnoleg (STS) sy'n edrych ar y modd y cawsant eu creu a'u datblygu.
    • Rydym yn astudio’r modd y mae gwyddoniaeth a thechnoleg yn eistedd o fewn eu cyd-destunau hanesyddol, diwylliannol a chymdeithasol.
    • Hynny yw, mae'r modd yr ydym yn gweld ac yn dehongli'r byd o'n cwmpas yn dibynnu ar argaeledd offer gwyddonol megis microsgopau, synwyryddion amgylcheddol, ac ati.
    • Felly, os yw gwyddoniaeth yn tueddu i atgynhyrchu braint a phersbectif grwpiau elitaidd, yna mae gwyddoniaeth dinasyddion yn cynnig y posibilrwydd o wyddoniaeth i'r bobl, gan y bobl.

Y Barri

    • Mae'n ymwneud â gwaith bio-màs yng nghanol y dref, y mae ymgyrchwyr amgylcheddol lleol yn dadlau y dylid ei ddymchwel.
    • Edrychon ar y modd y mae aelodau Grŵp Gwyddoniaeth Dinasyddion y Barri (BCSG) wedi ceisio atal y gwaith bio-màs rhag gweithredu.
    • Maent wedi treulio 15 mlynedd yn craffu ar y cyfreithiau a'r rheoliadau sydd wedi arwain at y penderfyniadau cynllunio a thrwyddedu hyd yn hyn.
    • Ein dadl yw bod y gwaith y maent wedi'i wneud hyd yn hyn yn rhagflaenydd hanfodol i waith casglu data mwy ffurfiol dan arweiniad y gymuned.

Jurassic Park yn 30 a'r chwyldro effeithiau arbennig ddigwyddodd yn sgil y ffilm

Retrieved on: 
Saturday, June 10, 2023
The Abyss, Land, Lost World, Industrial Light & Magic, Center, ILM, BBC, Walking with Dinosaurs, 3-Hydroxyasparagine, Helfa gelf, Fe, Close Encounters of the Third Kind, BYD, Lost Ark, Walking, Lost, HEB, Toyota Dyna, CGI, Raider, Journey to the Center of the Earth, OND, Public land mobile network, Entertainment, Cardboard, Creative industries, Dinosaur, Magic

Defnyddiodd Jurassic Park 1993 ddelweddau a gynhyrchwyd gan gyfrifiadur (CGI) arloesol i ddod â deinosoriaid yn fyw yn addasiad Steven Spielberg o'r nofel o'r un enw.

Key Points: 
  • Defnyddiodd Jurassic Park 1993 ddelweddau a gynhyrchwyd gan gyfrifiadur (CGI) arloesol i ddod â deinosoriaid yn fyw yn addasiad Steven Spielberg o'r nofel o'r un enw.
  • Daeth y ffilm yn ddigwyddiad yr oedd yn rhaid ei weld yn gyflym iawn a chafodd cynulleidfaoedd eu syfrdanu gan yr olygfa o weld deinosoriaid credadwy yn ymlwyybro ar draws y sgrin fawr am y tro cyntaf.
  • Nid yn unig y gwnaeth Jurassic Park gamau enfawr mewn gwneud ffilmiau effeithiau arbennig, ond fe wnaeth hefyd baratoi'r ffordd ar gyfer myrdd o gynyrchiadau dilynol a oedd yn cynnwys bwystfilod o bob lliw a llun.
  • Fe oedd awdur a chyfarwyddwr y ffilm, Westworld (1973), oedd yn adrodd stori parc adloniant lle’r oedd androidau yn camweithio ac yn rhedeg yn benwyllt.

CGI ac animeiddio

    • Darparodd Dennis Muren o’r cwmni effeithiau arbennig, Industrial Light and Magic (ILM), ymagwedd amgen drwy ddefnyddio modelu ac animeiddio CGI.
    • Ar gefn gwaith CGI arloesol yn The Abyss (1989) a Terminator 2: Judgement Day (1991), cynhyrchodd Muren a'i dîm gyfres brawf o ddeinosoriaid ysgerbydol.
    • Adeiladodd y dechneg hon fodel y deinosor o esgyrn, ychwanegu cyhyr ac yna yn olaf, y croen.
    • Er bod y darnau CGI yn gymharol fyr, maent yn cael effaith enfawr ar y stori, heb sôn am y gred bod y digwyddiad yn digwydd o'n blaenau mewn gwirionedd.

Effaith

    • Roedd hefyd yn gyfle perffaith i ddatblygu ac arddangos y datblygiadau diweddaraf mewn CGI.
    • Roedd y wefr o weld rhuthr y Gallimimus, arswyd ymosodiad y T. Rex ac arswyd yr helfa Velociraptor wedi swyno cynulleidfaoedd ar draws y byd.
    • Ond yn fwy na hynny, fe helpodd i greu chwyldro yn y defnydd o effeithiau arbennig CGI mewn ffilmiau.

FDA Provides Akebia Therapeutics a Path Forward for Vadadustat

Retrieved on: 
Tuesday, May 30, 2023
Mitsubishi Tanabe Pharma, Chronic kidney disease, Civil service commission, Symantec Endpoint Protection, Vadadustat, Rockwell Medical, Health care, Clinical trial, NDA, AKBA, Hepatotoxicity, Gross metropolitan product, Dialysis, European Commission, Division, Patient, European Economic Area, Risk, Anemia, OND, Medicines and Healthcare products Regulatory Agency, United Kingdom, Webcast, CRL, FDA, Letter, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, CKD, Complete Response Letter, Food, Kidney disease, Safety, Medical device, Pharmaceutical industry, VAT, Akebia

The CRL raised a concern regarding the increased risk of thromboembolic events, driven by vascular access thrombosis (VAT).

Key Points: 
  • The CRL raised a concern regarding the increased risk of thromboembolic events, driven by vascular access thrombosis (VAT).
  • Accordingly, Akebia plans to include post-approval data from Japan in the NDA resubmission, where tens of thousands of Japanese patients with CKD have been exposed to vadadustat to date.
  • The OND's letter provides guidance on a path for the resubmission of our NDA and potential approval of vadadustat for dialysis dependent patients in the U.S. without suggesting the need to generate additional clinical data.
  • We appreciate the FDA's engagement with us throughout the appeal process," said John P. Butler, Chief Executive Officer of Akebia.

Former FDA Senior Officials Join Greenleaf Health: Sandra Kweder, M.D., former Deputy Director of FDA’s Office of New Drugs & Donald D. Ashley, J.D., former Director of CDER’s Office of Compliance

Retrieved on: 
Thursday, February 2, 2023
Consulting, Biotechnology, FDA, Professional Services, White House, Federal Government, Health, General Health, Healthcare Reform, Pharmaceutical, Medical Devices, Public Policy, Government, Rear admiral, Science, Liaison, PDUFA, Center for Drug Evaluation and Research, Drug development, Division, HIV/AIDS, EMA, OND, Internal medicine, Food, U.S. Public Health Service reorganizations of 1966–1973, European Medicines Agency, Pregnancy, CDER, Public, Food and Drug Administration, China Biologic Products, Lactation, Regulatory compliance, Pharmaceutical industry, Medical device, Research, Policy

Kweder and Ashley each bring unique subject matter expertise that expands Greenleaf's unmatched regulatory advising capabilities.

Key Points: 
  • Kweder and Ashley each bring unique subject matter expertise that expands Greenleaf's unmatched regulatory advising capabilities.
  • Dr. Sandra Kweder is an internal medicine expert with more than 30 years of experience in U.S. and international medical products regulation and policy.
  • Prior to her work in Europe, Sandy spent nearly 14 years as the Deputy Director of CDER’s Office of New Drugs.
  • “It was difficult to leave FDA, so full of dynamic, intelligent, committed experts – I never had a dull moment in over 30 years.